BMRN 10-K & 10-Q changes, risk factors and insider trading

InWe 2024,continue to pursue the corporate strategy we announced ourin new2024, corporatewhich strategy foris focused on innovation, growth, and value commitment, which includes, among other things, the acceleration or discontinuation of certain programs, the continued expansion of our pipeline, updateupdates to our commercial organizational model, and cost transformation. If we are unable to successfully execute our strategy, our business, financial condition and results of operations may be materially and adversely affected.

As part of the strategy, we have announced that we are advancingcontinuing to advance VOXZOGO for the treatment of conditions beyond achondroplasia, including hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency. VOXZOGO addresses larger patient populations than most of our other products, and product candidates that we are currently developing or may license or acquire in the future may be intended for similarly larger patient populations than we have historically targeted. We are also expanding our pipeline through external innovation. In December 2025, we announced our proposed acquisition of Amicus Therapeutics, Inc. (Amicus) to expand and diversify our rare disease product portfolio (the Amicus Acquisition). In addition, in July 2025 we acquired Inozyme Pharma, Inc. (Inozyme) to strengthen our Enzyme Therapies portfolio, adding a late-stage enzyme replacement therapy, BMN 401 (formerly INZ-701). In order to continue the development of suchour product candidates and marketing of products with larger markets, we will need to continue expanding our operations. To manage expansion effectively, we need to continue to develop and improve our research and development capabilities, manufacturing and quality capacities, sales and marketing capabilities, financial and administrative systems and standard processes for global operations. For example, strong demand for VOXZOGO in certain markets outpaced our projections in the past, and we have previously faced challenges meeting demand despite sufficient materials. While supply constraints have eased and we expect to have the ability to meet estimated demand and support ongoing clinical programs, we may face challenges meeting demand in the future. Our staff, financial resources, systems, procedures or controls may be inadequate to support our operations and may increase our exposure to regulatory, competitive, and corruption risks and our management may be unable to manage successfully current or future market opportunities or our relationships with customers and other third parties.

Universities and public and private research institutions also compete with us. While these organizations primarily have educational or basic research objectives, they may develop proprietary technology and acquire patents that we may need for the development of our product candidates. We willhave in the past attempted and may in the future attempt to license this proprietary technology, if available. These licenses may not be available to us on acceptable terms, if at all. If we are unable to compete successfully with respect to acquisitions, joint venture and other collaboration opportunities, we may be limited in our ability to develop new products and to continue to expand our product pipeline.

We have had fewer interactions with regulatory authorities outside the U.S. and the EU as compared to our interactions with the FDA, the EC and the EMA. The approval procedures vary among countries and can involve additional clinical testing, and the time required to obtain approval may differ from that required to obtain FDA or EC approval. Moreover, clinical trials conducted in one country may not be accepted by regulatory authorities in other countries. Approval by the FDA or EC does not ensure approval by regulatory authorities in other countries, and approval by one or more non-U.S. regulatory authorities does not ensure approval by regulatory authorities in other non-U.S. countries or by the FDA or EC. However, a failure or delay in obtaining regulatory approval in one country may have a negative effect on the regulatory process in others. The non-U.S. regulatory approval process may include all of the risks associated with obtaining FDA or EC approval. We may not obtain non-U.S. regulatory approvals on a timely basis, if at all. We may not be able to file for regulatory approvals and even if we file, we may not receive necessary approvals to commercialize our product candidates in any market.

Regulatory authorities and the new requirements and guidelines they promulgate may lengthen the regulatory review process, require us to perform additional or larger studies, increase our development costs, lead to changes in regulatory positions and interpretations, delay or prevent approval and commercialization of our product candidates or lead to significant post-approval studies, limitations or restrictions. For example, on April 26, 2023, the EC adopted a proposal for a new Directive and Regulation to revise the existingEU pharmaceutical legislation.reform Whilewill discussions are still ongoing as part of the legislative process, this proposal maygenerally result in a decrease in data and market exclusivity for our product candidates in the EU.

In addition, some of our product candidates are intended to be used in combination with a medical device, such as an injector or other delivery system. Some of these products intended to be used with a medical device may be regulated as “combination products” in the U.S. and the EU, which are generally defined as products consisting of components from two or more regulatory categories (e.g., drug/device, device/biologic, drug/biologic). In the U.S., each component of a combination product is subject to the requirements established by the FDA for that type of component, whether a new drug, biologic or device. In order to facilitate pre-market review of combination products, the FDA designates one of its centers to have primary jurisdiction for the pre-market review and regulation of the overall product based upon a determination by the FDA of the primary mode of action of the combination product. The determination whether a product is a combination product or two separately regulated products is made by the FDA on a case-by-case basis. In the EU, medical devices and medicinal products are regulated separately, through different legislative instruments. The related applicable requirements will vary depending on the type of drug-device combination product. If, for example, a device intended to administer a medicinal product is sold together with such medicinal product in such a way that they form a single integral product which is intended exclusively for use in the given combination and which is not reusable, that single integral product is regulated as a medicinal product. In addition, the relevant general safety and performance requirements (GSPRs) established for medical devices by EU medical devices legislation apply to the device component of such combination products. In addition, some of our products require use with an in vitro companion diagnostic. Our product candidates may also require use with an in vitro companion diagnostic if the FDA determines that the companion diagnostic is essential for safe and effective use of the product candidate. The FDA generally will require approval or clearance of the diagnostic, known as a companion diagnostic, at the same time that the FDA approves the therapeutic product. Most companion diagnostics require approval of a premarket approval application. In the EU, companion diagnostics are deemed to be in vitro diagnostic medical devices and must conform with the applicable GSPRs. To demonstrate compliance with the GSPRs, companion diagnostics must undergo a conformity assessment by a Notified Body. If the related medicinal product has been, or is in the process of being, authorized through the centralized procedure for the authorization of medicinal products, the Notified Body will, before it can issue athe CErelevant CertificateEU oftechnical Conformity,documentation assessment certificate, be required to seek a scientific opinion from the EMA on the suitability of the companion diagnostic for use in relation to the medicinal product concerned. For medicinal products that have been or are in the process of authorization through any other route provided in EU legislation, the Notified Body must seek the opinion of the national competent authority of an EU Member State. Our product candidates intended for use with separately regulated devices, such as companion diagnostics, or expanded indications that we may seek for our products used with such devices, may not be approved or may be substantially delayed in receiving approval if the devices do not gain and/or maintain their own regulatory approvals, clearances, or certifications. Where approval of the drug or biologic product and device is sought under a single application, such as a drug with an injector or delivery system, the increased complexity of the review process may delay approval. The FDA and EU review processes and related criteria are complex, which could also lead to delays in the approval process. In addition, because these devices are provided by unaffiliated third-party companies, we are dependent on the sustained cooperation and effort of those third-party companies both to obtain regulatory approval and to maintain their own regulatory compliance. Failure of third-party companies to assist in the approval process or to maintain their own regulatory compliance could delay or prevent approval of our product candidates, or limit our ability to sell a product once it is approved.

Our marketed products have received regulatory approval to be commercially marketed and sold in the U.S., the EU, and certain other countries except ROCTAVIAN, which has received regulatory approval to be commercially marketed in the U.S. and conditional approval to be commercially marketed in the EU.countries. Any product for which we have obtained regulatory approval, or for which we obtain regulatory approval in the future, along with the manufacturing processes and practices, post-approval clinical research, product labeling, advertising and promotional activities for such product, are subject to continual requirements of, and review by, the FDA, the EC, the EMA and/or other comparable international and national regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, registration and listing requirements, current Good Manufacturing Practices (cGMP) requirements relating to manufacturing, quality control, quality assurance and corresponding maintenance of records and documents, import and export requirements and record keeping.

In addition, in the EU, the marketing authorization for BRINEURA was granted under “exceptional circumstances”. As a result, the risk-benefit balance of BRINEURA is reviewed annually and the marketing authorization may be withdrawn if the risk-benefit ratio is no longer favorable. The conditional marketing authorization for ROCTAVIAN is, moreover, valid for one year and must be reviewed annually until all related conditions have been fulfilled to permit transfer to a full authorization. Failure to continue to show favorable risk-benefit balance for BRINEURA or satisfy the conditions related to ROCTAVIAN’s conditional marketing authorization could result in the withdrawal of the marketing approvals for these products.approval.

Moreover, promotional communications with respect to prescription drugs, including biologics, are subject to a variety of legal and regulatory restrictions and must be consistent with the information in the product’s approved labeling and Summary of Product Characteristics. In particular, a product may not be promoted for uses that are not approved by the FDA or the EC as reflected in the product’s approved labeling. Although the FDA and other comparable international and national regulatory authorities do not regulate a physician’s choice of drug treatment made in the physician’s independent medical judgment, they do restrict promotional communications from companies or their sales force with respect to off-label uses of products for which marketing clearance has not been issued. The FDA and other national competent authorities or international regulatory authorities actively enforce the laws and regulations prohibiting the promotion of off-label uses, and a company that is found to have improperly promoted off-label uses may be subject to significant civil, criminal and administrative penalties. Thus, we are not able to promote any products we develop for indications or uses for which they are not approved. Additionally, in the EU, it is prohibited to promote prescription drugs to the general public and we are therefore limited to promote our products exclusively to healthcare professionals.professionals, which is also subject to restrictions. Public prosecutors, industry associations, healthcare professionals and other authorities and members of the public, including competitors, closely scrutinize advertising and promotion of any product in the EU.

We expect that pricing, coverage and reimbursement may be increasingly restricted in all the markets in which we sell our products. The escalating cost of healthcare has led to increased pressure on the healthcare industry to reduce costs. In particular, drug pricing by pharmaceutical companies has been under scrutiny for many years and continues to be subject to intense political and public debate in the U.S. and abroad. Governmental and private third-party payers have proposed healthcare reforms and cost reductions. A number of federal and state proposals to control the cost of healthcare, including the cost of drug treatments, have been made in the U.S. Specifically, there have been several U.S. congressional inquiries and proposed bills and enacted legislation designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, and reform government program reimbursement methodologies for drugs. Further, Congress and the executive branch have each indicated that they will continue to seek new legislative and/or administrative measures to control drug costs. In some international markets, the government controls the pricing, which can affect the profitability of drugs. Current government regulations and possible future legislation regarding healthcare may affect coverage and reimbursement for medical treatment by third-party payers, which may render our products not commercially viable or may adversely affect our future revenues and gross margins.

PriorOur industry is highly regulated and changes in law may adversely impact our business, operations or financial results. In the U.S., there have been and continue to be a number of legislative initiatives to contain healthcare costs. In the IRA,U.S., there have been several congressional inquiries, proposed and enacted federal and state legislation and executive action designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform government program reimbursement methodologies for drug products. Any reduction in reimbursement from Medicare and other government programs may result in a similar reduction in payments from private payers. For example, the Patient Protection and Affordable Care Act of 2010, as amended by the Health Care and Education Reconciliation Act of 2010 (collectively, the PPACA), expanded healthcare coverage within the U.S., primarily through the imposition of health insurance mandates on employers and individuals and expansion of the Medicaid program. Several provisions of the law have affected us and increased certain of our costs. Since its enactment, there have been executive, judicial and congressional challenges to certain aspects of the PPACA. Although the PPACA has generally been upheld thus far, it is unclear how continued challenges to the law may impact the PPACA and our business. In addition, other legislative changes have been adopted since the PPACA was enacted. Some of these changes have resulted in additional reductions in Medicare and other healthcare funding, which could have a material adverse effect on our customers and, accordingly, our financial operations.

In addition, individual states inAt the U.S. state level, legislatures have also increasingly enacted laws and implemented regulations designed to control pharmaceutical product pricing, including price or patient reimbursement constraints, price and price increase disclosure and reporting requirements, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing. Moreover, regional healthcare authorities and individual hospitals are increasingly using bidding procedures to determine what pharmaceutical products and which suppliers will be included in their prescription drug and other healthcare programs.

Likewise, in many EU Member States, legislators and other policymakers continue to propose and implement healthcare cost-containing measures in response to the increased attention being paid to healthcare costs in the EU. Certain of these changes could impose limitations on the prices we will be able to charge for our commercial products and any product candidates or the amounts of reimbursement available for these products from governmental and private third-party payers, may increase the tax obligations on pharmaceutical companies or may facilitate the introduction of generic competition with respect to our products. Further, an increasing number of EU Member States and other non-U.S. countries use prices for medicinal products established in other countries as “reference prices” to help determine the price of the product in their own territory. If the price of one of our products decreases substantially in a reference price country, it could impact the price for that product in other countries. Consequently, a downward trend in prices of our products in some countries could contribute to similar downward trends elsewhere, which would have a material adverse effect on our revenues and results of operations. Moreover, some EU Member States may require the completion of additional studies that compare the cost-effectiveness of a particular medicinal product candidate to currently available therapies. This Health Technology Assessment (HTA) process, which is currently governed by the national laws of the individual EU Member States, is the procedure according to which the assessment of the public health impact, therapeutic impact and the economic and societal impact of use of a given medicinal product in the national healthcare systems of the individual country is conducted. The outcome of HTA regarding specific medicinal products will often influence the pricing and reimbursement status granted to these medicinal products by the competent authorities of individual EU Member States. In 2022, the EC adopted the HTA regulation, which is intended to boost cooperation among EU Member States in evaluating new medicinal products. The HTA regulation becameentered applicableinto sforce in January 2025 and mayhas resultresulted in increased downward pricing pressure in the EU.

In the U.S., the company that first obtains FDA approval for a designated orphan drug for a given rare disease receives marketing exclusivity for use of that drug for the designated condition for a period of seven years. Orphan drug exclusive marketing rights may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantity of the drug. In addition, the FDA may approve another drug during a period of orphan drug exclusivity if the second drug is found to be clinically superior to the first drug. InUnder the current rules in the EU, a ten-year period of market exclusivity for the approved therapeutic indication (extendable to twelve years for orphan drugs that have complied with an agreed Pediatric Investigation Plan (PIP) pursuant to Regulation 1901/2006), during which the EC and EU Member States cannot accept another marketing authorization (MA) application or accept an application to extend existing authorizations for similar medicinal products for the same indication and no MA can be granted. MAs may also be granted to a similar medicinal product with the same orphan indication if: (i) the applicant can establish that the second medicinal product, although similar to the orphan medicinal product already authorized is safer, more effective or otherwise clinically superior to the orphan medicinal product already authorized; (ii) the MA holder for the first orphan medicinal product grants its consent; or (iii) if the MA holder of the orphan medicinal product is unable to supply sufficient quantities. MAs may also be granted for the same therapeutic indication in relation to products that are not similar. The period of market exclusivity may, in addition, be reduced to six years if, at the end of the fifth year, it can be demonstrated on the basis of available evidence that the criteria for its designation as an orphan medicine are no longer satisfied, for example if the original orphan medicinal product has become sufficiently profitable not to justify maintenance of market exclusivity. BecauseFurthermore, it is expected that these periods will be shortened as a result of a reform of the extentEU pharmaceutical legislative package. On December 11, 2025, the European Parliament and scopethe European Council reached a political agreement on the proposed revision of patentseveral European legislative instruments related to medicinal products, including orphan products. Among other things, the revision will amend the duration of the regulatory exclusivity. In particular, the regulatory data protection forperiod some(during which other companies cannot access product data) would amount to eight years, with one additional year of ourmarket protection (during which generic or biosimilar products iscannot limited,be orphansold), drugfollowing designationan andMA. resultingPharmaceutical regulatorycompanies exclusivitywould is especially important for our products that arebe eligible for orphanadditional drugperiods designation.of Formarket eligibleprotection products,under wecertain planconditions, towith relya cap of eleven years on the exclusivitycombined periodregulatory underprotection period. Orphan medicinal products addressing a disease with no current available medicinal treatment (“breakthrough orphan medicinal products”) would benefit from up to eleven years of market exclusivity. These rules, if formally approved by the OrphanEuropean Drug ActParliament and/or the OrphanEuropean Regulation, as applicable,Council to maintainbecome alaw, competitivecould position.adversely If we do not obtain orphan drug designation and related regulatory exclusivity foraffect our products that do not have broad patent protection or if a competing product is determined to be, for example, "clinically superior" to any of our products that has secured orphan drug exclusivity, our competitors may then sell the same drug to treat the same condition and our revenues will be reduced.products.

Even though we have obtained orphan drug designation for certain of our product candidates and even if we obtain orphan drug designation for our future product candidates, due to the uncertainties associated with developing biopharmaceutical products, we may not be the first to obtain marketing approval for any particular orphan indication, which means that we may not obtain orphan drug regulatory exclusivity and could also potentially be blocked from approval of certain product candidates until the competitor product’s orphan drug exclusivity period expires. Moreover, with respect to certain biologics and gene therapies,biologics, there may be some uncertainty regarding how similarity between product candidates designed to treat the same rare disease or condition may affect such product candidates’ orphan drug regulatory exclusivities. For biologics and gene therapies,biologics, the FDA’s determination of whether a drug is the same drug or a different drug will be based on the principal molecular structural features of the products. For gene therapy products, the FDA has stated in guidance that it generally intends to consider certain key features such as transgenes and vectors used in gene therapy products to be principal molecular structural features. The FDA has not yet proffered additional information on orphan drug sameness for gene therapy or similar products. Further, even if we obtain orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different drugs can be approved for the same condition and the same drug can be approved for different conditions and potentially used off-label in the orphan indication. The FDA could also interpret the term “condition” narrowly, which could allow for indirect competition during the period of exclusivity. Even after an orphan drug is approved and granted orphan drug exclusivity, the FDA can subsequently approve the same drug for the same condition if the FDA concludes that the later drug is saferclinically superior by means of greater safety or moreeffectiveness effectiveby or makesmaking a major contribution to patient care. OrphanFurther, orphan drug designation neither shortens the development time or regulatory review time of a drug, nor gives the drug any advantage in the regulatory review or approval process.

Our ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ, ROCTAVIANPALYNZIQ and VIMIZIM products are regulated by the FDA as biologics under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act (the PHS Act). Biologics require the submission of a Biologics License Application (BLA) and licensure by the FDA prior to being marketed in the U.S. The Biologics Price Competition and Innovation Act of 2009 (BPCIA) created a regulatory pathway under the PHS Act for the abbreviated licensure of biological products that are demonstrated to be “biosimilar” to or “interchangeable” with an FDA-licensed biological product. A similar abridged MA process is available to biosimilar products in the EU. In particular,the applicantsEU, fora MAsbiosimilar is typically defined as a biological medicine similar to another already approved biological medicine. Developers of biosimilars are required to demonstrate through comprehensive comparability studies withby the referencebest biologicalpossible medicinemeans that: a) their biological medicine is highly similar to the reference medicine,medicine in physicochemical and biological terms, notwithstanding natural variability inherent to all biological medicines; and b)that thereany observed differences are noduly clinicallyjustified meaningfulwith differencesregard betweento thetheir biosimilarpotential andimpact theon reference medicine in terms of safety, qualitysafety and efficacy.

Our products approved under BLAs in the U.S. or as a result ofU.S., Marketing Authorization Applications (MAAs) in the EU, or comparable regulatory approval applications in other countries, as well as our product candidates that may be approved in the future, could be reference products for biosimilar marketing applications. The FDA has been changing the requirements for demonstrating biosimilarity, which may make it easier for biosimilars referencing our product to be licensed. In the U.S., a follow-on biologic may be deemed interchangeable with, and automatically substitutable for, a reference product if its sponsor can demonstrate that the biosimilar product can be expected to produce the same clinical result as the reference product in any given patient, and for a product that is administered more than once, that the risk of switching in terms of safety or diminished efficacy of alternating or switching between the reference product and biosimilar product is not greater than the risk of maintaining the patient on the reference product. In the U.S., standards for interchangeability also are changing to make it easier for biosimilars to demonstrate interchangeability. Even though the BPCIA establishes a period of 12 years of data exclusivity for reference products, such data exclusivity only blocks licensure of biosimilars relying on the product as a reference product; it will not prevent the licensure of the same product for the same or different indications that does not seek to rely on reference product data. In the EU, a medicinal product containing a new active substance currently benefits from eight years of data exclusivity, during which biosimilar applications referring to the data of that product may not be accepted by the regulatory authorities, and a further two years of market exclusivity, during which biosimilar applications may be submitted and the reference product's data may be referenced but biosimilar products may not be placed on the market. The two-year period may be extended to three years if during the first eight years a new therapeutic indication with significant clinical benefit over existing therapies is approved.

ChangesDisruptions in funding forat the FDA, the EMA, other comparable regulatory authorities and other government agenciesagencies, including a reduction in some agencies' workforces and/or governmentinadequate shutdownsfunding, could hinder the ability of such authorities and agencies to hire and retain key leadership and other personnel or otherwise prevent those authorities and agencies from performing normal functions on which the operation of our business may rely, which could negatively impact our business.

ChangesDisruptions in funding levels ofat regulatory authorities and government agencies due to changes in funding levels, government shutdowns, reorganization, reduction in force or statutory, regulatory and policy changes can affect their ability to hire and retain key personnel and carry out their normal functions that support our business. For example, the current U.S. administration recently implemented or proposed policies, including substantial reductions to the FDA’s workforce, that may affect the FDA’s review process and hinder the ability of the FDA or the EMA to timely review and approve INDsregulatory or MAAssubmissions for our product candidates may be hindered by a lack of resources and qualified personnel.candidates. In addition, funding of other regulatory authorities and government agencies on which our operations rely, including those that fund research and development activities, is subject to the political budget process, which is inherently fluid and unpredictable.

The following Management’s Discussion and Analysis of Financial Condition and Results of Operations (MD&A) is intended to help the reader understand our results of operations and financial condition. The MD&A is provided as a supplement to, and should be read in conjunction with, our audited Consolidated Financial Statements and the accompanying notes to the Consolidated Financial Statements and other disclosures included in this Annual Report on Form 10-K, including the disclosures under “Risk Factors” in Part I, Item 1A of this Annual Report on Form 10-K. These risks and uncertainties could cause actual results to differ significantly from those projected in forward-looking statements contained in this report or implied by past results and trends. Forward-looking statements are statements that attempt to forecast or anticipate future developments in our business, financial condition or results of operations. See the section titled “Forward-Looking Statements” that appears at the beginning of this Annual Report on Form 10-K. These statements, like all statements in this report, speak only as of the date of this Annual Report on Form 10-K (unless another date is indicated), and, except as required by law, we undertake no obligation to update or revise these statements in light of future developments. Our Consolidated Financial Statements have been prepared in accordance with United States (U.S.) generally accepted accounting principles (GAAP) and are presented in U.S. Dollars (USD).

We are a leading, global rare disease biotechnology company dedicatedfocused toon translating the promise of genetic discovery intodelivering medicines thatfor makepeople aliving profoundwith impactgenetically ondefined the life of each patient.conditions. Our San Rafael, California-based company, founded in 1997, has a proven track record of innovation with eight commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we pursueseek treatmentsto unleash the full potential of genetic science by pursuing category-defining medicines that offerhave newa possibilitiesprofound forimpact patientson and families around the world navigating rare or difficult to treat genetic conditions.patients. A summary of our commercial products, as of December 31, 2024,2025, is provided below:

Conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions, could impact our global revenue sources and our overall business operations. The extent and duration of such effects remain uncertain and difficult to predict. We are actively monitoring and managing our response and assessing actual and potential impacts to our operating results and financial condition, as well as developments in our business, which could further impact the developments, trends and expectations described below. See the risk factor, “Our business is affected by macroeconomic conditions.” described in “Risk Factors” in Part I, Item 1A of this Annual Report on Form 10-K.

WeIn 2025, we achieved $3.2 billion in total revenues, including a significant contribution from our ongoing expansion of VOXZOGO, and we continued to grow our commercial business and advance our product candidate pipeline during 2024.pipeline. We believe that the combination of our internal research programs and partnerships and acquisitions of external assets will allow us to continue to develop and commercialize innovative therapies for people with serious and life-threatening rare diseases and medical conditions. We periodically conduct strategic portfolio assessment of research and development programs to determine which we believe have the strongest combination of scientific merit, opportunity for commercial success and potential value creation for stockholders. Based on such strategic portfolio assessments, certain programs that do not meet its threshold for further development and commercialization could be discontinued.

•VIMIZIM: higher sales volume due to new patients initiating therapy in the U.S. and timing of orders in countries that place large government orders, particularlyprimarily from countries in Europe;the Middle East and Latin America;

•ROCTAVIANBRINEURA: higher sales volume from new patients treatedinitiating therapy, primarily in the U.S. and Europe.Latin America.

The unfavorable impact of foreign currency exchange rates on USD reported results in 2025 was primarily driven by weakening of the Argentine Peso, Brazilian Real and Mexican Peso, partially offset by strengthening of the Euro. The unfavorable impact of foreign currency exchange rates on USD reported results in 2024 was primarily driven by the weakening of the Argentine Peso and the Japanese Yen.

Cost of Sales includes raw materials, personnel, facility and other costs associated with manufacturing our commercial products. These costs include production materials, production costs at our manufacturing facilities, third-party manufacturing costs, amortization of technology transfer intangible assets and internal and external final formulation and packaging costs. Cost of Sales also includes royalties payable to third parties based on sales of our products, idle plant costs and charges for inventory valuationwrite reserves.downs.

We continuously evaluate the recoverability of costs associated with pre-launch or pre-qualification manufacturing activities, if any, and capitalize the costs incurred related to those activities if we determine that recoverability is highly likelyprobable and therefore future revenues are expected. If the related product candidate's marketing application is rejected by the applicable regulators and the likelihood of future revenues for a product candidate become uncertain, the related manufacturing costs are expensed as R&D expenses.

The increase in G&A expense for 20242025 compared to 20232024 was primarily due to severance$118.5 andmillion of restructuring costscharges associatedrelated withto impairment of long-lived assets as a result of our portfoliostrategic strategy review and the associated organizational redesign efforts announceddecision in 2024, and increased bad debt expense during the fourth quarter of 2024.2025 to voluntarily withdraw ROCTAVIAN from the market, and partially due to incremental administrative costs related to ongoing support of business initiatives during the year. These increases were partially offset by bad debt expense recorded in the fourth quarter of 2024 that did not recur in 2025.

Amortization of intangible assets: the decrease in amortization expense for 20242025 as compared to 20232024 was due to the increase in the estimated useful life of an intangible asset as a result of the extension of a patent during the second half of 2024 and an intangible asset becoming fully amortized during the fourth quarter of 2023.2024.

The decrease in Interest Expense decreased in 20242025 as compared to 2023 was2024 primarily due to the August 1, 2024 maturitysettlement of our convertible debt (the 2024 Notes). that matured in August 2024. We expect Interest Expense to decreaseincrease over the next 12 months due to financing related to the settlementpending ofAmicus ouracquisition, 2024including Notes.the 2034 Notes issued on February 12, 2026. See Note 1020 to our accompanying Consolidated Financial Statements for additional information regarding ourfinancing debt.related to the pending acquisition of Amicus.

Other Expense,Income (Expense), Net

Other Expense,Income (Expense), Net for the periods presented was as follows:

The decreaseincrease in Other Expense,Income (Expense), Net, in 20242025 compared to 20232024 was primarily due to theproceeds lowerfrom foreigninsurance currencyrelated transactionto lossesdamaged and decreased loss on non-marketable securities.goods.

Certain countries in which we have operations, including Ireland, have adopted Pillar Two rules,framework, recently released from the Organisation for Economic Co-operation and Development (OECD), including a minimum tax rate of 15%. ItThe isU.S. uncertainhas whethernot the United States will enactenacted legislation to adopt the Pillar Two framework. The adoption of the Pillar Two framework did not have a material impact on our effective tax rate and we plan to continue evaluating additional guidance released by the OECD, along with the pending legislative adoption by additional individual countries.

We believe cash generated from sales of our commercial products, in addition to our cash, cash equivalents and short-term investmentsinvestments, including proceeds from the 2034 Notes and external financings, will be sufficient to satisfy our liquidity requirements for at least the next 12 months.months, including our agreement to acquire Amicus in an all-cash transaction. We believe we will meet longer-term expected future cash requirements and obligations through a combination of cash flows from operating activities and available cash and long-term investment balances. We will need to raise additional funds by issuing equity, debt or convertible securities, taking loans or entering into collaborative or other agreements if we are unable to satisfy our liquidity requirements. For example, we may require additional financing to fund the repayment of our convertibleoutstanding debt due in 2027,indebtedness, future milestone payments and our future operations, including the commercialization of our products and product candidates currently under development, preclinical studies and clinical trials, and potential licenses and acquisitions. The timing and mix of our funding alternatives could change depending on many factors, including how much we elect to spend on our development programs, potential licenses and acquisitions of complementary technologies, products and companies or if we settle our convertible debt in cash. In addition, depending on prevailing market conditions, our liquidity requirements, contractual restrictions, and other factors, we may also from time to time seek to retire or purchase our outstanding debt through cash purchases and/or exchanges for equity securities, in open market purchases, privately negotiated transactions or otherwise.

We are mindful that conditions in the current macroeconomic environment, such as inflation, changes in interest and foreign currency exchange rates, natural disasters, geopolitical instability, impact of new or increased tariffs and escalating trade tensions, regulatory uncertainty, and supply chain disruptions could affect our ability to achieve our goals. In addition, we sell our products in certain countries that face economic volatility and weakness. Although we have historically collected receivables from customers in such countries, sustained weakness or further deterioration of the local economies and currencies may cause customers in those countries to be unable to pay for our products. We will continue to monitor these conditions and will attempt to adjust our business processes, as appropriate, to mitigate macroeconomic risks to our business.

The increase in net cash provided by operating activities in 20242025 compared to 20232024 was primarily attributed to theincrease improvedin operatingnet performanceincome adjusted for non-cash items mainly related to inventory write-off and timingasset ofimpairments cashfor receiptsROCTAVIAN and IPR&D charges from ourInozyme customers,acquisition, and partially offsetdue byto the timing of payments to vendors,vendors increasedand personnel-relatedcash payments resultingreceipts from our ongoing organizational redesign efforts and payments of income taxes.customers.

The increase in net cash used in financinginvesting activities in 20242025 compared to 20232024 was primarily dueattributable to the $495.0$285.2 million settlementnet cash paid for the acquisition of theInozyme, 2024lower Notes,net whichmaturities maturedof inavailable-for-sale Augustsecurities, 2024.and higher purchases of property, plant and equipment.

Our $600.0 million (undiscounted) of total convertible debt as of December 31, 20242025 will impact our liquidity due to the semi-annual cash interest payments as well as the repayment of the principal amount, if not converted. As of December 31, 2024, our indebtedness consistedconsisting of our 1.250%1.25% senior subordinated convertible notes due in 2027 (the 2027 Notes), which, if not converted, will beimpact requiredour liquidity due to besemi-annual repaidcash interest payments and repayment of the principal amount in cash at maturity in May 2027.2027 if not converted.

InOur August$600.0 2024, we entered into anmillion unsecured revolving credit facility providing(Revolving forFacility) $600.0as millionof inDecember revolving31, loan commitments. The credit facility2025 is intended to finance ongoing working capital needs and for other general corporate purposes.initiatives. The creditRevolving facilityFacility matures in August 2029 and contains financial covenants including a maximum total net leverage ratio and a minimum interest coverage ratio. The credit facility matures in August 2029. As of December 31, 20242025 there were no amounts outstanding under the creditRevolving facilityFacility and we were in compliance with all covenants. The New Revolving Facility is expected to replace the existing Revolving Facility.

As of December 31, 2024,2025, we had purchase obligations of approximately $641.9$590.8 million, of which $482.0$354.1 million is expected to be paid in 2025.2026. Our purchase obligations are primarily related to firm purchase commitments entered into in the normal course of business to procure active pharmaceutical ingredients, certain inventory-related items, certain third-party R&D services, production services and facility construction services. The amount also includes hosting fees and other enterprise resource planning (ERP) system implementation costs for which we are committed.

We continue to pursue theOur corporate strategy we announced in 2024, which is focused on innovation, growth, and value commitment, which includes, among other things, the acceleration or discontinuation of certain programs, the continued expansion of our pipeline, updates to our commercial organizational model, and cost transformation. If we are unable to successfully execute our strategy, our business, financial condition and results of operations may be materially and adversely affected.

As part of the strategy, we are continuing to advance VOXZOGO for the treatment of conditions beyond achondroplasia, including hypochondroplasia, idiopathic short stature, and Noonan syndrome. VOXZOGO addresses larger patient populations than most of our other products, and product candidates that we are currently developing or may license or acquire in the future may be intended for similarly larger patient populations than we have historically targeted. We are also expanding our pipeline through external innovation. InFor example, in April 2026, we acquired Amicus Therapeutics, Inc. (Amicus) to expand and diversify our rare disease product portfolio (the Amicus Acquisition). In addition, in July 2025 we acquired Inozyme Pharma, Inc. (Inozyme) to strengthen our Enzyme Therapies portfolio, adding a late-stage enzyme replacement therapy, BMN 401 (formerly INZ-701). In order to continue the development of our product candidates and marketing of products with larger markets, we will need to continue expanding our operations. To manage expansion effectively, we need to continue to develop and improve our research and development capabilities, manufacturing and quality capacities, sales and marketing capabilities, financial and administrative systems and standard processes for global operations. Our staff, financial resources, systems, procedures or controls may be inadequate to support our operations and may increase our exposure to regulatory, competitive, and corruption risks and our management may be unable to manage successfully current or future market opportunities or our relationships with customers and other third parties.

As part of our new corporate strategy, we have acquired, entered into agreements to acquire, and may continue to acquire, companies or businesses that we believe could complement, expand or enhance our product offerings. For example, in April 2026, we acquired Amicus to expand and diversify our rare disease product portfolio.portfolio Inand addition,acquired Inozyme Pharma, Inc. in July 2025, we completed the acquisition of Inozyme2025 to strengthenadd oura Enzymelate-stage Therapiesenzyme portfolioreplacement withtherapy product candidate, BMN 401, which is currently being assessed for the treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1401 (ENPP1formerly INZ-701) deficiency.. Our acquisition strategy may divert the attention of management and cause us to incur various expenses in identifying, investigating and pursuing suitable acquisitions, whether or not such acquisitions are consummated.

In addition, once an acquisition is closed, integrating our business practices and operations with the acquired business’ so that we can fully realize the anticipated benefits of the acquisition could require us to devote significant management attention and resources. The success of current and future acquisitions will depend, in part, on our ability to realize the anticipated benefits from successfully combining our and the acquired businesses’ operations. We have in the past and may in the future face risks or experience difficulties successfully integrating acquired businesses, such as Inozyme or Amicus, with our operations. Such difficulties could result in the failure to achieve revenue that we anticipate, the loss of key employees that may be difficult to replace in the very competitive pharmaceutical field, the failure to harmonize both companies’ corporate cultures, the disruption of each company’s ongoing businesses or inconsistencies in standards, controls, procedures and policies that adversely affect our ability to maintain relationships with suppliers, collaboration partners, clinical trial investigators or managers of our clinical trials. See “—Risks Related to the Amicus Acquisition” in this section for additional risks related to the Amicus Acquisition.

Acquisitions could also result in dilutive issuances of equity securities, the incurrence of debt, contingent liabilities, amortization expenses, impairment of goodwill and/or purchased long-lived assets, and restructuring charges, any of which could adversely affect our operating results and financial condition. For example, we have incurred significant indebtedness in connection with the Amicus Acquisition. In addition, as experienced with BMN 401, acquired product candidates,candidates suchhave not and may not perform as BMN 401,expected, may not result in regulatory approval, may not perform as expected, may not be successful, may require significantly greater resources and investments than originally anticipated or may not produce the revenues, earnings or business synergies that we anticipated. As a result, the anticipated benefits of an acquisition may not be realized fully within the expected timeframe or at all or may take longer to realize or cost more than expected, which could materially and adversely affect our business, financial condition, results of operations and growth prospects.

Our competitors may develop, manufacture and market products that are more effective or less expensive than ours. They may also obtain regulatory approvals for their products faster than we can obtain them (including those products with orphan drug designation, which may prevent us from marketing our product entirely for seven years, along with other regulatory exclusivities that could block approval) or commercialize their products before we do. With respect to VOXZOGO, we face competition from other companies that have developed, and may in the future develop, products for treatment for achondroplasia. In addition, we face competition with respect to GALAFOLD and POMBILITI + OPFOLDA from several large pharmaceutical and biotechnology companies that currently market and sell products for the treatment of Fabry disease and Pompe disease. As we commercialize our products, we have faced and may continue to face intense competition from other pharmaceutical companies, some of which may have more extensive resources and/or established relationships in the communities we seek to treat. If we do not compete successfully, our revenues would be adversely affected, and we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product.

We also face competition from generic versions of our products. For example, generic versions of KUVAN are available in several countries around the world, including in the U.S. and the European Union (EU), which has adversely affected and will continue to adversely affect our revenues from KUVAN. In addition, we have and may in the future face challenges outside of the U.S. from competitors seeking to develop generic versions of GALAFOLD. Competitors launching generic versions of our products independently establish the price of such products and determine the types of discounts or rebates they will offer parties that purchase or pay for the product. Generic competition often results in decreases in the net prices at which branded products can be sold. After any introduction of a generic product, a significant percentage of the prescriptions written for our branded products will likely be filled with the generic product. Certain U.S. state laws allow for, and in some instances in the absence of specific instructions from the prescribing physician mandate, the dispensing of generic products rather than branded products when a generic version is available. We expect that the approval and launch of generic versions of our products and the approval and launch of branded products that compete with our products will continue to have a negative impact and could have a material adverse effect on our sales of our products and on our business, financial condition, results of operations and growth prospects.

*We have in the past entered and may in the future enter into licensing arrangements, and we may not realize the benefits of such licensing arrangements.

We have in the past entered and may in the future enter into licensing arrangements with third parties.parties, either directly or through an acquisition, such as Amicus’ collaboration agreement with respect to GALAFOLD. It is possible that we may not achieve financial or strategic benefits that justify a specific license, or we may otherwise not realize the benefits of such licensing arrangement. Further, licensing arrangements impose various diligence, milestone and royalty payment and other obligations on us. If we fail to comply with our obligations under any current or future licenses, our licensors may have the right to terminate these license agreements, which could harm our business prospects, financial condition and results of operations. Additionally, counterparties to our license agreements have in the past alleged and may in the future allege that we have breached a license agreement, which can result in litigation or other disputes that can divert management’s attention away from our business and require us to expend resources, as well as potentially having to negotiate new or reinstated licenses with less favorable terms. Any such situation could adversely affect our business, financial condition, and results of operations.

The success of the Amicus Acquisition will depend, in part, on our ability to realize the anticipated benefits from successfully combining our and Amicus’ businesses. We have and plan onto devotingcontinue to devote substantial management attention and resources to integrating our and Amicus’ businesses so that we can fully realize the anticipated benefits of the Amicus Acquisition. This integration process may be disruptive to our and Amicus’ businesses, and, if implemented ineffectively, could restrict realization of the expected benefits of the Amicus Acquisition. In addition, the acquired Amicus business, including GALAFOLD and POMBILITI + OPFOLDA, may not be successful, may require greater resources and investments than originally anticipated or may result in the assumption of unknown or contingent liabilities, which could have an adverse effect on us or our results of operations and our financial guidance.

•preserving the important licensing, marketing, supply, distribution, and other commercial relationships of Amicus;

Any of these risks could adversely affect our ability to maintain relationships with collaboration partners, vendors, employees and other commercial relationships or adversely affect our future operational results. As a result, the anticipated benefits of the Amicus Acquisition may not be realized fully or at all or may take longer to realize or cost more than expected, which could adversely affect our business, financial condition, including our ability to generate sufficient cash to service our indebtedness, including our 1.25% senior subordinated convertible notes due in 2027 (the 2027 Notes), our 5.5% senior unsecured notes due in 2034 (the 2034 Notes and together with the 2027 Notes, the Notes) and any loans outstanding under the 2026 Credit Agreement (as defined below), results of operations, growth prospects and our financial guidance, and we may be forced to take other actions to satisfy our obligations under our indebtedness, which may not be successful. In addition, changes in laws and regulations could adversely impact our business, financial condition, results of operations and growth prospects. Furthermore, the risks discussed above could also adversely affect our ability to accurately forecast the operational and financial performance of the combined company, including key financial metrics and prospects. If we are unable to accurately forecast and meet our financial guidance, our stock price can be materially adversely affected.

*We may not realize the anticipated cost savings from the Amicus Acquisition.

The benefits that we expect to achieve as a result of the Amicus Acquisition will depend, in part, on our ability to realize anticipated cost savings, including resulting from expected operational synergies and global infrastructure efficiencies. AfterFollowing the closing of the Amicus Acquisition, we believe that we will be able to, among other matters, save on our costs by being able to streamline administrative processes and harmonize global distribution of the two companies.

Our success in realizing these cost savings, and the timing of this realization, depends on many factors. Even ifAlthough we arehave able to consummateconsummated the Amicus Acquisition successfully,Acquisition, this may not result in the full realization of the cost savings that we currently expect, either within the expected timeframe, or at all. In addition, we cannot assure you that the costs to achieve these cost savings will not be higher than we anticipated. Therefore, we cannot assure you that any anticipated cost savings will be achieved or that our estimates and assumptions will prove to be accurate. If our cost savings are less than our estimates or our costscost savings initiatives adversely affect our business or cost more or take longer to implement than we project, or if our assumptions prove to be inaccurate, our results could be lower than we anticipate.

*Our actual financial positions and results of operations may differ materially from the publicly filed unaudited pro forma condensed combined financial information.

The unaudited pro forma condensed combined financial information contained in our amended Current Report on Form 8-K/A filed on JanuaryJuly 26,13, 2026 is presented for illustrative purposes only and may differ materially from what our actual financial position or results of operations would have been had the Amicus Acquisition been completed on the dates indicated. The unaudited pro forma condensed combined financial information was derived from our and Amicus’ audited and unaudited historical financial statements and certain adjustments and assumptions have been made regarding the combined company after giving effect to the Amicus Acquisition and related transactions. The assets and liabilities of Amicus were measured at fair value based on various preliminary estimates using assumptions that we believed were reasonable utilizing information currently available. The process for estimating the fair value of acquired assets and assumed liabilities requires the use of judgment in determining the appropriate assumptions and estimates. These estimates may be revised as additional information becomes available and as additional analyses are performed. Differences between preliminary estimates in the unaudited pro forma condensed combined financial information and the final acquisition accounting will occur and could have a material impact on the unaudited pro forma condensed combined financial information and the ultimate combined company’s financial position and future results of operations.

In addition, the assumptions used in preparing the unaudited pro forma condensed combined financial information may not prove to be accurate, and other factors may affect our financial condition or results of operations following the completionclosing of the Amicus Acquisition.Acquisition in April 2026.

*Any product for which we have obtained regulatory approval, or for which we obtain approval in the future, is subject to, or will be subject to, extensive ongoing regulatory requirements by the FDA, the EC, the EMA and other comparable international regulatory authorities, and if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, we may be subject to penalties, we will be unable to generate revenues from the sale of such products, our potential for generating positive cash flow will be diminished, and the capital necessary to fund our operations will be increased.

Our marketed products have received regulatory approval to be commercially marketed and sold in the U.S., the EU, and certain other countries. Any product for which we have obtained regulatory approval, including the products acquired in the Amicus Acquisition, or for which we obtain regulatory approval in the future, along with the manufacturing processes and practices, post-approval clinical research, product labeling, advertising and promotional activities for such product, are subject to continual requirements of, and review by, the FDA, the EC, the EMA and/or other comparable international and national regulatory authorities. These requirements include submissions of safety and other post-marketing information and reports, registration and listing requirements, current Good Manufacturing Practices (cGMP) requirements relating to manufacturing, quality control, quality assurance and corresponding maintenance of records and documents, import and export requirements and record keeping.

Moreover, if original FDA approval for one of our product candidates is granted via the accelerated approval pathway, we will be required to conduct a post-marketing confirmatory trial to verify and describe the clinical benefit in support of full approval. An unsuccessful post-marketing study or failure to complete such a study with due diligence could result in the withdrawal of the FDA’s marketing approval for a product candidate. For example, GALAFOLD and VOXZOGO isare approved in the U.S. under accelerated approval basedand onare ansubject improvementto inpost-marketing annualizedrequirements growthto velocity.obtain full approval. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. To fulfill this post-marketing requirement, we intend to use our ongoing open-label extension studies compared to available natural history. In addition, the FDA and the EC often require post-marketing testing and surveillance to monitor the effects of products. The FDA, the EC and other comparable international regulatory authorities may condition approval of our product candidates on the completion of such post-marketing clinical studies. These post-marketing studies may suggest that a product causes undesirable side effects or may present a risk to the patient.

*We may face competition from biosimilars approved through an abbreviated regulatory pathway.

Our ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQPALYNZIQ, VIMIZIM and VIMIZIM productsPOMBILITI are regulated by the FDA as biologics under the Federal Food, Drug, and Cosmetic Act and the Public Health Service Act (the PHS Act). Biologics require the submission of a Biologics License Application (BLA) and licensure by the FDA prior to being marketed in the U.S. The Biologics Price Competition and Innovation Act of 2009 (BPCIA) created a regulatory pathway under the PHS Act for the abbreviated licensure of biological products that are demonstrated to be “biosimilar” to or “interchangeable” with an FDA-licensed biological product. A similar abridged MA process is available to biosimilar products in the EU. In the EU, a biosimilar is typically defined as a biological medicine similar to another already approved biological medicine. Developers of biosimilars are required to demonstrate by the best possible means that their biological medicine is highly similar to the reference medicine in physicochemical and biological terms, notwithstanding natural variability inherent to all biological medicines; and that any observed differences are duly justified with regard to their potential impact on safety and efficacy.

Disruptions at regulatory authorities and government agencies due to changes in funding levels, government shutdowns, reorganization, reduction in force or statutory, regulatory and policy changes can affect their ability to hire and retain key personnel and carry out their normal functions that support our business. For example, the current U.S. administration recentlypreviously implemented or proposed policies, including substantial reductions to the FDA’s workforce, that may affect the FDA’s review process and hinder the ability of the FDA to timely review and approve regulatory submissions for our product candidates. In addition, funding of other regulatory authorities and government agencies on which our operations rely, including those that fund research and development activities, is subject to the political budget process, which is inherently fluid and unpredictable.

As of MarchJune 31,30, 2026, we had cash and cash equivalents totaling $2.2 billion, restricted cash equivalents of $850.0$874.0 million and debt obligations of $1.5$4.3 billion (undiscounted), which consisted of $1.5 billion under the Notes. In April 2026, we incurredNotes, $2.0 billion of indebtedness under a senior secured term loan B facility (the Term Loan B Facility) and $800.0 million of indebtedness under a senior secured term loan A facility (the Term Loan A Facility and, together with the Term Loan B Facility, the Term Facilities). In April 2026, we incurred indebtedness under the Term Facilities and entered into a $600.0 million senior secured revolving credit facility (the 2026 Revolving Facility and, together with the Term Facilities, the 2026 Senior Secured Credit Facilities.Facilities). The 2027 Notes, if not converted, will be required to be repaid in cash at maturity in May 2027. We will need cash not only to pay the ongoing interest due on the Notes and under the credit agreement governing the 2026 Senior Secured Credit Facilities (the 2026 Credit Agreement), but also to repay the principal amounts associated with the 2027 Notes (if not converted), the 2034 Notes and any Loans (as defined therein) outstanding under the 2026 Credit Agreement.

In addition, the indenture governing our 2034 Notes and the 2026 Credit Agreement contain, and any future indebtedness that we may incur may contain, financial and/or other restrictive covenants that limit our ability to operate our business, raise capital or make payments under our other indebtedness. If we fail to comply with these covenants or to make payments under our indebtedness when due, then we would be in default under that indebtedness, which could, in turn, result in that and our other indebtedness becoming immediately payable in full. If we default under the 2026 Credit Agreement or the indentures governing our Notes, the outstanding borrowings thereunder could become immediately due and payable, the 2026 Revolving Facility lenders could refuse to permit additional borrowings under the 2026 Revolving Facility, or it could lead to defaults under agreements governing our current or future indebtedness, including the 2026 Credit Agreement and the indentures governing the Notes, as applicable

As of MarchJune 31,30, 2026, our outstanding indebtedness consisted of the 2027 Notes, which, if not converted, will be required to be repaid in cash at maturity in May 2027 and2027, the 2034 Notes, which will be required to be repaid in cash at maturity in February 2034.2034, and the Term Facilities. While we could seek to obtain additional third-party financing to pay for any amounts due in cash upon maturity of the Notes, we cannot be sure that such third-party financing will be available on commercially reasonable terms, if at all. If not sooner repaid, loans outstanding under our Term Loan B Facility will be required to be repaid in cash at maturity in April 2033 and loans outstanding under our Term Loan A Facility and 2026 Revolving Facility will be required to be repaid in cash at maturity in April 2031.

*If we are unable to successfully develop and maintain manufacturing processes for our product candidates to produce sufficient quantities at acceptable costs, we may be unable to support a clinical trial or be forced to terminate a program, or if we are unable to produce sufficient quantities of our products at acceptable costs, we may be unable to meet commercial demand, lose potential revenue, have reduced margins or be forced to terminate a program.

WeIn addition, we currently rely on third parties for all or portions of the manufacture of each of our products. For example, GALAFOLD and POMBILITI are manufactured by single-source third-party manufacturers. If those manufacturers are unwilling or unable to fulfill their contractual obligations or satisfy demand outside of or in excess of the contractual obligations, we may be unable to meet demand for these products or sell these products at all and we may lose potential revenue. Further, the availability of suitable contract manufacturing capacity at scheduled or optimum times is not certain.

A significant and growing portion of our revenues and earnings, as well as our substantial international assets and liabilities, are exposed to changes in foreign exchange rates. As we operate in multiple foreign currencies, including the Euro, the Brazilian Real, the Japanese Yen, the Canadian Dollar, the Argentine Peso, the British Pound, the Colombian Peso, the Mexican Peso, the British PoundPeso and several other currencies, changes in those currencies relative to the U.S. Dollar (USD) have in the past and may in the future impact our revenues and expenses. If the USD were to weaken against another currency (as has been the case against the Euro in 2025), assuming all other variables remained constant, our revenues would increase, having a positive impact on earnings, and our overall expenses would increase, having a negative impact on earnings. Conversely, if the USD were to strengthen against another currency (as was the case for many currencies in 2022), assuming all other variables remained constant, our revenues would decrease, having a negative impact on earnings, and our overall expenses would decrease, having a positive impact on earnings. In addition, because our financial statements are reported in USD, changes in currency exchange rates between the USD and other currencies have had, and will continue to have, an impact on our results of operations. Therefore, significant changes in foreign exchange rates can impact our results and our financial guidance.

The ongoing trade tensions between the United States and other jurisdictions have resulted in multiple rounds of tariffs and anticipated tariffs affecting pharmaceuticals and pharmaceutical ingredients, including finished drug products, manufacturing equipment, and related supplies. Such tariffs may significantly increase our costs for certain products. The Bureau of Industry and Security, U.S. Department of Commerce, has initiated an investigation to determine whether pharmaceutical ingredients, including finished drug product, manufactured outside the United States pose a national security risk and should be subject to additional tariffs. Following that investigation, the President announced a proclamation which will impose 100% tariffs on certain patented pharmaceutical products and associated pharmaceutical ingredients.ingredients beginning July 31, 2026, unless modified by the President. We are continuing to assessmonitor the potential impact of the proclamation and any enabling regulatory actions on our business. Unlike consumer goods, pharmaceuticals face unique regulatory constraints that make rapid supply chain adjustments particularly difficult and costly. Since we conduct manufacturing and packaging operations in Ireland for certain of our products and also engage contract manufacturers around the world, the import of our products into the United States is subject to tariffs. Notwithstanding the U.S. Supreme Court's recent decision invalidating tariffs imposed under the International Emergency Economic Powers Act, the magnitude and impact of tariffs are uncertain and are subject to a variety of factors, including the effective date and duration of additional tariffs, changes in the amount, scope and nature of tariffs in the future, including as a result of litigation or other challenges, any retaliatory tariffs that other countries may impose in response to tariffs levied by the United States and any mitigating actions that may become available. Trade restrictions and export regulations, or increases in tariffs and additional taxes, including any retaliatory measures, could negatively impact demand, increase our supply chain complexity and our manufacturing costs, decrease margins, reduce the competitiveness of our products, or restrict our ability to sell products, provide services or purchase necessary equipment and supplies, any or all of which could have a material and adverse effect on our business, results of operations, or financial condition. In addition, the dynamic and unpredictable tariff and trade landscape creates substantial uncertainty and significant planning challenges for our operations and complicates our long-term investment decisions regarding manufacturing facilities, supply chain optimization, and research and development locations.

We are a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined conditions. OurFounded in 1997, our San Rafael, California-based company, founded in 1997,company has a proven track record of innovation, with a portfolio ofnine commercial therapies and a strong clinical and preclinical pipeline. Using a distinctive approach to drug discovery and development, we seek to unleash the full potential of genetic science by pursuing category-defining medicines that have a profound impact on patients.

A summary of our commercial products, as of MarchJune 31,30, 2026, is provided below:

(1) In 2026, we announced that we will no longer market ROCTAVIAN. For additional information related to ROCTAVIAN, see Note 19 - Restructuring to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2025.

The increase in Net Product Revenues for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was primarily attributed to the following:

•VOXZOGO: higher sales volume from new patients initiating therapy across all regions.regions; and

•ALDURAZYMEVIMIZIM: lower sales volume due to timing of orderlarge fulfillmentgovernment toorders Sanofioutside the U.S.; and

In certain countries, governments place large periodic orders for our products. We expect that the timing of these large government orders will continue to be inconsistent, which has created in the past and may continue to create significant period to period variation in our revenues. We expect Total Revenues to increase over the next 12 months following the Amicus acquisition that closed in April 2026.

With respect to VOXZOGO, GALAFOLD and POMBILITI + OPFOLDA, see also the risk factors “Our success depends on our ability to manage our growth and execute our corporate strategy.” and “If we fail to compete successfully with respect to product sales, we may be unable to generate sufficient sales to recover our expenses related to the development of a product program or to justify continued marketing of a product and our revenues could be adversely affected.” in "Risk Factors" in Part II, Item 1A of this Quarterly Report for additional information on risk factors that could impact our business and operations.

We face exposure to movements in foreign currency exchange rates, and use foreign currency exchange forward contracts to hedge a percentage of our foreign currency exposure, primarily the Euro. Certain currencies are not included in our hedging program, such as the Argentine Peso. With respect to the risks posed by fluctuations of both hedged and unhedged currencies against the U.S. dollar (USD), see the risk factor “Our international operations pose currency risks, which may adversely affect our operating results and net income” in “Risk Factors” included in Part II, Item 1A of this Quarterly Report for additional information. The following table shows our Net Product Revenues denominated in USD and foreign currencies:

The favorable impact for the three and six months ended MarchJune 31,30, 2026 was primarily driven by strengthening of the Euro and Mexican Peso,Euro, partially offset by weakening of the Argentine Peso and the Japanese Yen.Peso. The unfavorable impact for the three and six months ended MarchJune 31,30, 2025 was primarily driven by weakening of the Argentine Peso, Brazilian Real, Euro, Colombian PesoReal and ArgentineMexican Peso.

Cost of Sales includes raw materials, personnel and facility and other costs associated with manufacturing our commercial products. These costs include production materials, production costs at our manufacturing facilities, third-party manufacturing costs, amortization of technology transfer intangible assets and internal and external final formulation and packaging costs. Cost of Sales also includes royalties payable to third parties based on sales of our products, idle plant costs andcosts, charges for inventory write downs.downs and amortization of acquired inventory fair value step-up recorded from business combinations.

Cost of Salessales increased and Gross Marginmargin decreased in the three months ended MarchJune 31,30, 2026 as compared to the three months ended MarchJune 31,30, 2025, primarily due to an increase in sales volume and amortization of acquired inventory fair value step-up associated with the products acquired from Amicus. Cost of sales increased and Gross margin decreased in the six months ended June 30, 2026 as compared to the six months ended June 30, 2025, primarily due to an increase in sales volume, a $31.0 million charge recorded in first quarter of 2026 associated with an unsuccessful process qualification campaign to expand NAGLAZYME manufacturing capabilities.capabilities, and amortization of acquired inventory fair value step-up.

The increase in R&D expense for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was primarily due to higher spend on BMN 401, a later-stage clinical program acquired in the third quarter of 2025.2025, and an increase in R&D spend for two marketed products acquired from Amicus in April 2026. These increases were partially offset by lower spend on Research and early pipeline due to discontinued programs.

The increase in S&M expense for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was primarily due to increasedexpenses spendingassociated with commercial products acquired in the Amicus transaction and higher spend related to global expansion of Enzyme Therapies and VOXZOGO.

The increase in G&A expense for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was primarily due to integration and restructuring charges related to the acquisition of Amicus and incremental administrative costs related to ongoing support of corporate initiatives and pre-close costs related to Amicus acquisition.initiatives.

The increase in Amortization of Intangibleintangible Assetsassets for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was relativelyprimarily flat.due Weto the amortization of the intangible assets acquired from Amicus on April 27, 2026. With the addition on the acquired intangible assets, we expect Intangibleamortization Assetof Amortizationintangible assets to significantly increase over the next 12 months following the Amicus acquisition that closed in April 2026.months.

The increasedecrease in Interest Incomeincome for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was mainlyprimarily due to higher levels of cash,lower cash equivalents and investment balances.balances Theas $850.0investments millionwere proceedsliquidated fromto fund a portion of the issuanceAmicus of 2034 Notes in the first quarter of 2026 were held in escrow and invested in money market funds as of March 31, 2026.transaction. We expect Interest Incomeincome to decrease over the next 12 months due to lower cash and investment balances following the Amicus acquisition that closed in April 2026.balances.

The increase in Interest Expenseexpense for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was primarily due to issuanceborrowings obtained to finance a portion of 2034the NotesAmicus for $850.0 million aggregate principaltransaction in FebruaryApril 2026,2026. andThe $5.3increase millionwas ofalso partially attributable to bridge commitment fees recognizedthat duringwere amortized to interest expense following the threetermination monthsof endedthe Marchbridge 31, 2026.commitment. We expect Interest Expenseexpense to significantly increase over the next 12 months due to thelong-term financingdebt relatedfinancings to the Amicus acquisition that closedcompleted in April 2026, including the new term loans issued in April 2026. See Note 67 and- Note 13Debt to our accompanying Condensed Consolidated Financial Statements for additional information regarding our debt.

Other Income (Expense),Income, Net

Other Income (Expense),Income, Net for the periods presented was as follows:

The increase in Other Income (Expense),Income, Net for the three months ended MarchJune 31,30, 2026 as compared to the three months ended MarchJune 31,30, 2025 was relatively flat. The increase in Other Income, Net for the six months ended June 30, 2026 as compared to the six months ended June 30, 2025 was primarily due to gain on sale of marketable securities resulting from the sale of our short-term and long-term investments to fund the Amicus acquisition that closed in April 2026.transaction.

The decrease in Provision for Income Taxes for the three and six months ended MarchJune 31,30, 2026 as compared to the three and six months ended MarchJune 31,30, 2025 was primarily due to lower pre-tax income, partially offset by higher tax expense related to stock based compensation.income.

Our cash, cash equivalents, restricted cash equivalents and investments as of MarchJune 31,30, 2026 and December 31, 2025 were as follows:

We believe cash generated from sales of our commercial products, in addition to our cash, cash equivalents and restricted cash equivalents and external financings, will be sufficient to satisfy our liquidity requirements for at least the next 12 months, including our debt service commitments relating to the Amicus acquisition. We believe we will meet longer-term expected future cash requirements and obligations through a combination of cash flows from operating activities and available cash. We will need to raise additional funds by issuing equity, debt or convertible securities, taking loans or entering into collaborative or other agreements if we are unable to satisfy our liquidity requirements. For example, we may require additional financing to fund the repayment of our outstanding indebtedness, future milestone payments and our future operations, including the commercialization of our products and product candidates currently under development, preclinical studies and clinical trials, and potential licenses and acquisitions. The timing and mix of our funding alternatives could change depending on many factors, including how much we elect to spend on our development programs, potential licenses and acquisitions of complementary technologies, products and companies or if we settle our long-term debt in cash. In addition, depending on prevailing market conditions, our liquidity requirements, contractual restrictions, and other factors, we may also from time to time seek to retire or purchase our outstanding convertible debt through cash purchases and/or exchanges for equity securities, in open market purchases, privately negotiated transactions or otherwise.

The increase in net cash provided by operating activities in the threesix months ended MarchJune 31,30, 2026 compared to MarchJune 31,30, 2025 was primarily attributed to decrease in inventory campaigns and timing of cash receipts from our customers,customers and a decrease in inventory purchases, partially offset by a decrease in netNet income.Income.

The increase in net cash providedused byin financinginvesting activities in the threesix months ended MarchJune 31,30, 2026 compared to MarchJune 31,30, 2025 was primarily attributable to proceedsapproximately from$5.1 thebillion issuancenet cash paid for acquisition of 2034Amicus, Notes.partially Theoffset proceedsby fromhigher thenet 2034 Notes were held in escrow asmaturities of Marchmarketable 31, 2026 and subsequently used to finance a portion of the Amicus acquisition that closed in April 2026.securities.