Like Vertex Pharmaceuticals, but focused on a broader spectrum of rare genetic and metabolic diseases.

Amgen, but exclusively dedicated to developing therapies for rare genetic and metabolic disorders.

• Vimizim: An enzyme replacement therapy used to treat mucopolysaccharidosis (MPS) IV type A.
• Naglazyme: A recombinant enzyme for patients with mucopolysaccharidosis (MPS) VI.
• Kuvan: A proprietary synthetic oral form of 6R-BH4 used to treat patients with phenylketonuria (PKU).
• Palynziq: A PEGylated recombinant enzyme delivered via subcutaneous injection to reduce blood phenylalanine concentrations.
• Brineura: A recombinant human enzyme for the treatment of patients with ceroid lipofuscinosis type 2 (Batten disease).
• Voxzogo: A once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia.
• Aldurazyme: A purified protein designed to be identical to a naturally occurring human enzyme alpha-L-iduronidase.
• Valoctocogene Roxaparvovec: An adeno-associated virus vector gene therapy in Phase III clinical trial for severe hemophilia A.
• BMN 307: An AAV5 mediated gene therapy in Phase 1/2 clinical trial for normalizing blood phenylalanine concentration levels in patients with PKU.
• BMN 255: A therapy in Phase 1/2 clinical trial for treating primary hyperoxaluria.

Major Customers of BioMarin Pharmaceutical (BMRN)

BioMarin Pharmaceutical primarily sells its therapies to various organizations and commercial entities that then distribute or administer the products to patients. Its major customers include:

• Specialty pharmacies
• Hospitals
• Non-U.S. government agencies
• Distributors
• Pharmaceutical wholesalers

Alexander Hardy, President and Chief Executive Officer

Alexander Hardy joined BioMarin as President and Chief Executive Officer in December 2023. Before joining BioMarin, he served as the Chief Executive Officer of Genentech, Roche’s research and development arm, for four and a half years.

Brian R. Mueller, Executive Vice President and Chief Financial Officer

Brian R. Mueller joined BioMarin in December 2002 and currently serves as Executive Vice President and Chief Financial Officer, overseeing all Finance and Information Management teams. His previous roles at BioMarin include Senior Vice President, Finance and Chief Accounting Officer, and Group Vice President, Corporate Controller. Prior to BioMarin, Mr. Mueller worked for KPMG as a senior manager in the firm's audit practice, and before that, spent seven years with Arthur Andersen LLP in their audit and business advisory services practice. From June 2014 to May 2020, he was a member of the board of directors and served as Chairman of the Audit Committee for Anthera Pharmaceuticals, Inc., a previously public biopharmaceutical company.

Greg Friberg, Executive Vice President, Chief Research & Development Officer

Greg Friberg assumed the role of Executive Vice President, Chief Research & Development Officer, effective September 30, 2024. He joined BioMarin from Amgen, Inc., where he spent nearly two decades in various leadership positions, most recently as Vice President of Global Medical Affairs for their rare disease drug portfolio. Dr. Friberg brings extensive experience in clinical development and lifecycle management, having been responsible for advancing numerous medicines from investigational new drug application filing through late-stage development.

Cristin Hubbard, Executive Vice President and Chief Commercial Officer

Cristin Hubbard joined BioMarin in May 2024 as Executive Vice President and Chief Commercial Officer, leading the company's global commercial operations and portfolio strategy. She has over 20 years of experience in the biopharmaceutical and diagnostics industries. Most recently, she was the Head of Global Product Strategy for Roche Pharma, where she was responsible for lifecycle management and accelerating the delivery of medicines from development to commercialization across five therapeutic areas. During her more than 16 years at Roche and Genentech, Ms. Hubbard held various leadership positions, including global head of Diagnostics Partnering for Roche Diagnostics, focusing on licensing deals and acquisitions. She began her career as a medicinal chemist at Theravance.

C. Greg Guyer, Executive Vice President and Chief Technical Officer

C. Greg Guyer, Ph.D., joined BioMarin in May 2020 and currently serves as Chief Technical Officer and Executive Vice President of Global Manufacturing and Technical Operations. In this role, he is responsible for manufacturing, process development, quality, supply chain, engineering, and analytical chemistry departments. From 2015 to 2019, Dr. Guyer held several increasingly responsible positions at Bristol Myers Squibb (BMS), primarily leading all operations and biologic development. Before BMS, he worked for Merck & Co., Inc. from 1994 to 2015, where he led various global organizations in biologic and pharmaceutical operations, quality, regulatory, emerging markets strategy, and enterprise systems. He started his career at the FDA in the lab and rose to Division Director for Generic Drugs over 10 years.

The key risks for BioMarin Pharmaceutical (BMRN) include:

1. Clinical Development and Regulatory Approval of Gene Therapies: BioMarin has made substantial investments in its gene therapy pipeline, notably with valoctocogene roxaparvovec, an adeno associated virus vector in Phase III clinical trials for severe hemophilia A, and BMN 307, an AAV5 mediated gene therapy in Phase I/II for phenylketonuria (PKU). The successful development, manufacturing, and obtaining regulatory approval for these complex and novel gene therapies are critical for the company's future growth. Failures in late-stage clinical trials, unexpected safety concerns, or significant delays in regulatory processes could severely impact BioMarin's financial performance and long-term prospects.
2. Patent Expiry and Generic/Biosimilar Competition: BioMarin's revenue is largely derived from its portfolio of commercialized therapies for rare diseases. As these products mature, they become vulnerable to patent expiration, which can lead to the entry of generic or biosimilar competitors. For instance, Kuvan, a proprietary synthetic oral form of 6R-BH4 for PKU, is an older commercial product that could face increased competition as its market exclusivity periods conclude. The loss of patent protection for key revenue-generating products could result in substantial revenue erosion.
3. Market Access, Pricing Pressure, and Competition for Commercialized Rare Disease Therapies: Despite operating in the rare disease space, BioMarin's existing commercial products, such as Vimizim, Naglazyme, Palynziq, Brineura, Voxzogo, and Aldurazyme, face ongoing challenges related to market access, reimbursement negotiations, and pricing scrutiny from healthcare payers globally. Additionally, while these therapies address niche patient populations, the rare disease market can still experience competition from new entrants, or the development of alternative or improved treatments could erode the market share and profitability of BioMarin's current product portfolio.

BioMarin Pharmaceutical Inc. develops and commercializes therapies for serious and life-threatening rare diseases and medical conditions. The addressable markets for its main products and services are detailed below:

• Vimizim (mucopolysaccharidosis (MPS) IV type A): The global market for Morquio A syndrome therapies, for which Vimizim is the only FDA-approved biologic, is limited by the rarity of the disease. The estimated global Morquio A patient population is between 1,000 and 2,000 patients. The global Morquio Syndrome (MPS-IV) drug market size was US$ 1.9 million in 2025 and is expected to reach US$ 4.4 million by 2032.
• Naglazyme (MPS VI): The global market for Naglazyme is estimated at approximately $300 million annually. In 2023, Naglazyme sales were approximately $303 million.
• Kuvan and Palynziq (phenylketonuria (PKU)): The global phenylketonuria (PKU) market size was valued at USD 1.17 billion in 2023 and is estimated to reach USD 2.02 billion by 2032. Another source indicates the global Phenylketonuria Treatment Market size is estimated to be valued at USD 1.2 billion in 2025 and is expected to reach USD 2.1 billion by 2032. In 2023, the Phenylketonuria Market Size was approximately USD 700 million, with the United States accounting for about 60% of the total market size across the 7 Major Markets (7MM).
• Brineura (ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease): The global Batten disease drug pipeline market is projected to reach $60.65 million by 2025. The 7 major Batten disease markets are expected to exhibit a compound annual growth rate (CAGR) of 5.34%.
• Voxzogo (achondroplasia): The market size of achondroplasia in the 7MM is estimated to be around USD 300 million in 2024, with projections to grow to approximately USD 1.5 billion by 2034. Another report states the 7 major achondroplasia markets reached a value of USD 136.8 million in 2024 and are expected to reach USD 655.5 million by 2035. BioMarin estimates the potential market could encompass up to 420,000 patients across indications globally. For hypochondroplasia, an expansion for Voxzogo, BioMarin estimates a total addressable global population of about 14,000 diagnosed patients.
• Aldurazyme (MPS I): The Mucopolysaccharidosis I Market in the 7MM was valued at approximately USD 145 million in 2024 and is projected to grow at a CAGR of 8.0% from 2025 to 2034. In 2024, the MPS I market size in the U.S. was around USD 70 million, accounting for 48% of the total 7MM market.
• valoctocogene roxaparvovec (severe hemophilia A): Market size information for this specific product or the severe hemophilia A gene therapy market was not available in the search results.
• BMN 307 (PKU): This product is in clinical trials, and its addressable market is covered by the overall Phenylketonuria (PKU) market detailed above for Kuvan and Palynziq.
• BMN 255 (primary hyperoxaluria): The Primary Hyperoxaluria (PH) Treatment Market in the 7MM was approximately USD 316 million in 2023. The global primary hyperoxaluria treatment market reached US$ 168.96 million in 2023 and is expected to reach US$ 365.49 million by 2031. The United States accounted for approximately USD 281 million of the 7MM market in 2023, representing about 89% of that total.

BioMarin Pharmaceutical Inc. (BMRN) expects its future revenue growth over the next 2-3 years to be driven by several key factors:

1. Continued Growth of Voxzogo: Voxzogo, a treatment for achondroplasia, is anticipated to be a significant growth driver. The company expects sustained high patient demand and deeper market penetration, particularly in international markets. Voxzogo achieved 26% year-over-year revenue growth in 2025 and is projected to see high single-digit growth rates in 2026, with anticipated sales reaching $975 million to $1.025 billion. Further expansion is expected from potential approval for additional indications, such as hypochondroplasia, with Phase 3 data expected in the first half of 2026.
2. Sustained Growth in Enzyme Therapies: BioMarin's portfolio of enzyme therapies, including products like Naglazyme, Vimizim, Palynziq, and Aldurazyme, consistently demonstrates strong performance. This segment grew by 9% in 2025, with Palynziq showing robust 22% growth. BioMarin forecasts enzyme therapies revenue to be between $2.225 billion and $2.275 billion in 2026, driven by high market penetration and patient adherence across its extensive global footprint.
3. Acquisition of Amicus Therapeutics: The acquisition of Amicus Therapeutics, expected to close in the second quarter of 2026, is a key strategic move to bolster BioMarin's revenue. This acquisition will add high-growth products Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease to BioMarin's commercial portfolio. This is anticipated to provide a meaningful uplift to BioMarin's total revenue growth rate and become significantly accretive starting in 2027. An adolescent label expansion for Pombiliti + Opfolda is also anticipated in the second half of 2026.
4. Advancement of Pipeline Candidates: BioMarin has several promising pipeline candidates that could contribute to future revenue growth. These include BMN 401 for ENPP1 deficiency, which is expected to have Phase 3 data read out in the first half of 2026, and BMN 333, a long-acting CNP for achondroplasia currently in a Phase 2/3 study. The company continues to invest in innovation and advance its clinical-stage assets, indicating potential for new product launches or expanded indications in the coming years.

Share Repurchases

• BioMarin Pharmaceutical has not engaged in significant share repurchase programs over the last 3-5 years, with the latest available data indicating a negative or null share buyback ratio.

Share Issuance

• The number of BioMarin Pharmaceutical's common shares issued and outstanding saw a minor increase, reaching 192,300,091 shares by the end of 2025, up from 190,761,349 shares.

Outbound Investments

• In December 2025, BioMarin entered a definitive agreement to acquire Amicus Therapeutics for $4.8 billion in an all-cash transaction, expected to close in the second quarter of 2026. This acquisition is intended to accelerate and diversify BioMarin's revenue streams by adding therapies for Fabry and Pompe diseases.
• The company completed the acquisition of Inozyme Pharma in July 2025 for $270 million, which added BMN 401 for ENPP1 Deficiency to its pipeline.

Capital Expenditures

• BioMarin's annual capital expenditures were $119.2 million in 2021, $131.5 million in 2022, $107.6 million in 2023, and $97.4 million in 2024.
• Projected capital expenditures are $176 million for 2025, $193 million for 2026, and $211 million for 2027.
• These capital expenditures are primarily focused on supporting investment in innovation and future growth initiatives.