Here are 1-3 brief analogies for BioMarin Pharmaceutical (BMRN):

• Vertex Pharmaceuticals, but focused on a wider variety of rare genetic diseases.
• Alexion Pharmaceuticals, specializing in a portfolio of treatments for diverse rare genetic disorders.
• Amgen, but exclusively dedicated to developing therapies for rare genetic disorders.

BioMarin Pharmaceutical (BMRN) major products:

• Naglazyme (elosulfase alfa): An enzyme replacement therapy used to treat Mucopolysaccharidosis VI (MPS VI).
• Kuvan (sapropterin dihydrochloride): A medication used to treat phenylketonuria (PKU) in certain patients.
• Vimizim (elosulfase alfa): An enzyme replacement therapy for patients with Mucopolysaccharidosis IVA (MPS IVA), also known as Morquio A Syndrome.
• Palynziq (pegvaliase-pqpz): An enzyme substitution therapy for adults with phenylketonuria (PKU) who have uncontrolled blood phenylalanine levels.
• Voxzogo (vosoritide): A C-type natriuretic peptide analog used to increase linear growth in children with achondroplasia.
• Roctavian (valoctocogene roxaparvovec): A gene therapy for the treatment of severe hemophilia A in adults.

BioMarin Pharmaceutical (BMRN) sells primarily to other companies within the pharmaceutical distribution chain, rather than directly to individual patients. Like most pharmaceutical manufacturers, BioMarin relies on a concentrated group of large wholesale distributors to get its products to pharmacies, hospitals, and clinics.

Based on their financial filings, BioMarin's major customers are typically the three largest pharmaceutical wholesale distributors in the United States:

• Cencora (COR) - Formerly AmerisourceBergen.
• Cardinal Health, Inc. (CAH)
• McKesson Corporation (MCK)

These companies act as intermediaries, purchasing BioMarin's products and then distributing them to healthcare providers and pharmacies nationwide, who in turn dispense them to patients.

null

Alexander Hardy, President & Chief Executive Officer

Alexander Hardy became President and Chief Executive Officer of BioMarin in December 2023. Prior to joining BioMarin, he spent nearly two decades at Genentech and Roche, where he most recently served as Chief Executive Officer of Genentech. During his tenure as Genentech's CEO, he oversaw the successful launch of 10 innovative medicines and led the company's entry into several new therapeutic areas.

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

Brian R. Mueller joined BioMarin in December 2002 and currently serves as Executive Vice President, Chief Financial Officer, responsible for managing all Finance and Information Management teams. He previously held roles as Senior Vice President, Finance and Chief Accounting Officer, and Group Vice President, Corporate Controller. Before joining BioMarin, Mr. Mueller worked for KPMG as a senior manager in the firm's audit practice, joining KPMG after Arthur Andersen LLP ceased operations in June 2002, where he had spent seven years in audit and business advisory services. From June 2014 to May 2020, he served on the board of directors of Anthera Pharmaceuticals, Inc., a public biopharmaceutical company, where he also chaired the Audit Committee.

Greg Friberg, Executive Vice President, Chief Research Development Officer

Dr. Greg Friberg was appointed Executive Vice President, Chief Research Development Officer, effective September 30, 2024. Dr. Friberg brings nearly two decades of industry experience, having previously served as Vice President, Global Medical Affairs, Rare Disease at Amgen, Inc. During his 18 years at Amgen, he was responsible for advancing multiple medicines from investigational new drug application filing through late-stage development and headed Global Development for the company's hematology/oncology and bone portfolios.

Jeff Ajer, Executive Vice President, Chief Commercial Officer

Jeff Ajer serves as the Executive Vice President, Chief Commercial Officer of BioMarin Pharmaceutical Inc. He joined BioMarin in 2005 as one of the first sales and marketing employees and has held roles of increasing responsibility, including Vice President of Sales and Marketing Operations. Mr. Ajer has over 25 years of experience in commercializing rare diseases and specialty medicines, leading global marketing and sales organizations. Prior to BioMarin, he served as Vice President, Global Transplant Operations at Genzyme Corporation, and held positions at SangStat Medical Corporation and ICN Pharmaceuticals. During his time as CCO at BioMarin, he had direct responsibility for the launch of 5 brands.

G. Eric Davis, Executive Vice President, General Counsel & Secretary

G. Eric Davis is the Executive Vice President, General Counsel & Secretary at BioMarin Pharmaceutical. Further detailed background information beyond his title and current role at BioMarin was not readily available in the provided search results.

The key risks to BioMarin Pharmaceutical (BMRN) are:

1. Increasing Competition: BioMarin faces significant and increasing competition in the rare disease market, particularly for its flagship product, Voxzogo. New therapies from competitors like Ascendis Pharma (TransCon CNP) and BridgeBio Pharma (infigratinib) pose a direct threat to Voxzogo's market share in achondroplasia. This competitive pressure can erode market share, impact profitability, and lead to pricing pressures.
2. Reliance on a Limited Number of Products and Pipeline Dependency: The company's revenue is heavily dependent on a limited number of products. Its future growth is significantly tied to the successful development and commercialization of pipeline candidates, such as BMN333 and BMN351. Any unexpected issues with existing products, such as safety concerns or regulatory hurdles, or setbacks, delays, or failures in the development of key pipeline assets, could have a substantial impact on BioMarin's financial performance and long-term prospects.
3. Regulatory and Research & Development (R&D) Challenges: The development and commercialization of BioMarin's pharmaceutical products are subject to stringent regulations and approvals from global regulatory authorities. The company faces inherent risks associated with high R&D costs, potential clinical trial failures, and regulatory hurdles that can lead to program discontinuations or extended timelines. Additionally, the risk of patent expirations for key products like Vimizim and Palynziq could pressure margins.

1. Emerging Competition in Hemophilia A Gene Therapy: Pfizer's gene therapy, Beqvez (fidanacogene elaparvovec), received conditional marketing authorization in the European Union in February 2024 and is under review by the U.S. Food and Drug Administration. This represents a direct competitive threat to BioMarin's Roctavian (valoctocogene roxaparvovec) in the severe hemophilia A gene therapy market, particularly given Roctavian's already challenging commercial launch and slow uptake.

2. Emerging Competition in Achondroplasia Treatment: Ascendis Pharma's investigational therapeutic, TransCon CNP, is in Phase 2/3 clinical development for achondroplasia. As a long-acting C-type natriuretic peptide (CNP) analog with a different mechanism of action, TransCon CNP could emerge as a significant alternative treatment, posing a competitive threat to BioMarin's Voxzogo (vosoritide) in the achondroplasia market if it demonstrates superior efficacy, safety, or convenience.

3. Emerging Competition in Phenylketonuria (PKU) Treatment: Synlogic's SYNB1934, an investigational oral synthetic biotic, is currently in Phase 3 clinical development for the treatment of phenylketonuria. A successful launch of SYNB1934 could offer a novel and potentially more convenient or effective treatment option for PKU, thereby posing a clear competitive threat to BioMarin's existing PKU therapies, including Palynziq (pegvaliase).

Below are the addressable market sizes for BioMarin Pharmaceutical's main products:

• Voxzogo (vosoritide): Treats achondroplasia.
  • Addressable Patient Population: 24,000 patients globally.
  • Potential Market Expansion: The potential market could encompass up to 420,000 patients across various indications, with peak sales for the company's skeletal conditions business expected to exceed $5 billion.
  • Region: Global, with the U.S. being the largest single market opportunity, and markets outside the U.S. representing approximately 90% of eligible patients. BioMarin anticipates Voxzogo to be accessible in over 60 countries by 2027.
  • 2025 Sales Projection: BioMarin projects Voxzogo sales to be in the range of $900 million to $950 million for 2025.
• Brineura (cerliponase alfa): Treats neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as Batten disease.
  • Addressable Market Size: Null (specific global monetary market size not provided in the search results).
  • Region: Global, with North America being the largest revenue contributor. Asia-Pacific is identified as an emerging growth opportunity.
• Palynziq (pegvaliase-pqpz): Treats phenylketonuria (PKU).
  • Addressable Market Size: The global phenylketonuria treatment market size was estimated at USD 518.7 million in 2024 and is projected to reach USD 851.0 million by 2030. The global phenylketonuria market size is estimated to reach $2.02 billion by 2032.
  • Region: Global, across the seven major markets (7MM), which include the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. North America held the largest share in 2024, and Asia-Pacific is anticipated to be the fastest-growing region.
• Naglazyme (galsulfase): Treats mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy syndrome.
  • Addressable Market Size: Null (specific global monetary market size not provided in the search results).
  • Region: Global, with North America being the largest region in the Naglazyme market in 2024.
• Vimizim (elosulfase alfa): Treats Morquio A Syndrome (MPS IVA).
  • Addressable Patient Population: Approximately 3,000 patients in the developed world, with about 800 patients in the U.S.
  • Addressable Market Size: Analysts expected it to generate between $400 million and $500 million annually as of 2014.
  • Region: Global, with North America being the largest region in 2024.
• Roctavian (valoctocogene roxaparvovec-rvox): Gene therapy for severe hemophilia A.
  • Addressable Market Size: The broader hemophilia market was valued at about $14 billion in 2023 and is expected to double by 2030. Leerink Partners analysts once projected $2.2 billion in peak sales for Roctavian. The global gene therapy market size stood at USD 3.43 billion in 2024 and is projected to rise to USD 13.83 billion by 2032.
  • Region: BioMarin has narrowed its commercialization efforts for Roctavian to the U.S., Germany, and Italy.

Expected Drivers of Future Revenue Growth for BioMarin Pharmaceutical (BMRN)

BioMarin Pharmaceutical (BMRN) is anticipated to drive future revenue growth over the next 2-3 years through several key strategies focusing on its existing product portfolio and pipeline advancements:

• Continued Global Expansion and Label Enhancements for VOXZOGO: VOXZOGO, a treatment for achondroplasia, is a primary driver of BioMarin's revenue growth. The company expects sustained growth from its ongoing global launch and market expansion efforts. Furthermore, plans to expand VOXZOGO's label to include other growth disorders are anticipated to significantly boost future revenue. In the third quarter of 2024, VOXZOGO generated $190 million in revenue, marking a 54% increase year-over-year. BioMarin aims for VOXZOGO to generate the majority of its projected $4 billion revenue target by 2027.
• Growth of the Enzyme Therapies Portfolio: BioMarin's enzyme therapies, which include products such as ALDURAZYME, PALYNZIQ, BRINEURA, NAGLAZYME, and VIMIZIM, are expected to continue their strong performance. This growth is driven by increasing patient demand across all regions, focused initiatives, geographical expansions, and patient adherence programs. The enzyme therapies portfolio contributed $509 million in revenue in Q3 2024, a 27% increase from the previous year, with ALDURAZYME experiencing a 40% year-over-year revenue growth in 2024 due to order timing. PALYNZIQ is also noted for its double-digit revenue increases throughout 2024, with solid growth anticipated into 2025.
• Advancements and Launches from a Robust Pipeline: BioMarin's pipeline, particularly in skeletal conditions and enzyme therapies, is poised to contribute to future revenue. The company is anticipating two FDA approvals by 2027 and aims for 11 new product launches by 2034. Key pipeline advancements include late-stage clinical trials for VOXZOGO in hypochondroplasia and BMN 333, which could expand the company's leadership in skeletal disorders and create new revenue streams. The company expects to commence a Phase 2/3 study for BMN 333 in the first half of 2026 and anticipates VOXZOGO hypochondroplasia data in 2026.
• Strategic Acquisitions and Partnerships: BioMarin has identified strategic acquisitions and partnerships as priorities to accelerate growth and diversify its portfolio. The company has capital earmarked for acquiring assets that can be scaled by its global infrastructure. For example, the acquisition of Inozyme is expected to contribute to the company's robust growth.

Share Repurchases

• BioMarin used approximately $50.0 million of the net proceeds from a convertible notes offering in May 2020 to repurchase shares of its common stock concurrently with the offering.

Share Issuance

• In May 2020, BioMarin priced an upsized $550.0 million aggregate principal amount of 1.25% senior subordinated convertible notes due 2027, which was later increased to $600 million through the exercise of an option by initial purchasers.
• The number of common shares issued and outstanding has shown a slight increase, from approximately 181.7 million at the end of 2021 to around 190.8-192.0 million by the third quarter of 2025.

Outbound Investments

• BioMarin completed the acquisition of Inozyme in July 2025.
• The acquisition of Inozyme Pharma was for approximately $270 million, announced in May 2025.
• Acquired In-Process Research & Development (IPR&D) charges of $221 million were recorded through Q3 2025 related to the Inozyme Pharma acquisition.

Capital Expenditures

• BioMarin's annual capital expenditures were $137.5 million in 2020, $119.2 million in 2021, $131.5 million in 2022, $107.611 million in 2023, and $97.418 million in 2024.
• Projected capital expenditures are $176 million for 2025, $193 million for 2026, and $211 million for 2027.
• These capital expenditures support the company's priority of investment in innovation and future growth.