BioMarin Pharmaceutical

What BioMarin Pharmaceutical does

BioMarin Pharmaceutical is a publicly traded global rare-disease biotechnology company founded in 1997 and headquartered in San Rafael, California. The company develops and commercializes nine approved therapies for genetically defined conditions spanning skeletal disorders (VOXZOGO/vosoritide for achondroplasia and, pending label expansion, hypochondroplasia), lysosomal storage disorders (Vimizim for MPS IVA, Naglazyme for MPS VI, Aldurazyme for MPS I, Brineura for CLN2), and phenylketonuria (Palynziq, Kuvan). Its core technology platforms are enzyme replacement therapies, gene therapies, and C-type natriuretic peptide (CNP) analogs, with the CNP platform generating over 4,500 patient-years of clinical and real-world data. Following the April 2026 close of the $4.8 billion all-cash acquisition of Amicus Therapeutics, BioMarin added Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease, while voluntarily withdrawing Roctavian (hemophilia A gene therapy) from the U.S. market in March 2026 after weak uptake. The pipeline also includes BMN 333 (long-acting CNP analog, Phase 1) and BMN 401 (ENPP1 deficiency, Phase 3).

BioMarin generates revenue from direct sales of its commercial therapies, distributed through specialty pharmacies and site-of-care centers across approximately 80 countries. Pricing is specialty-pharmacy-driven and varies by geography, patient weight, disease severity, and insurance coverage, with no publicly disclosed list price. The company also receives royalties on licensed products (Kuvan and the withdrawn Roctavian), though Kuvan royalties are declining and Roctavian royalties ceased with the 2026 withdrawal. BioMarin reached $3.22 billion in FY2025 revenue (+13% YoY) and issued FY2026 guidance of $3.825–$3.925 billion, representing approximately 20% growth at the midpoint including Amicus. R&D remains funded through debt financing, including $850 million in senior notes and approximately $3.7 billion in non-convertible debt raised in early 2026 to fund the Amicus transaction.

The go-to-market combines enterprise field sales targeting rare-disease specialists and academic medical centers, community-led engagement through patient advocacy groups (National MPS Society since 2018, RARE Scholars, Accelerate Change Together initiative), and direct-to-consumer patient support programs such as PKU & ME. BioMarin deploys a multi-channel marketing approach across LinkedIn, X, Instagram, Facebook, YouTube, and TikTok, alongside peer-reviewed publications, medical conferences (ENDO 2026, ACMG, Pediatric Endocrine Society), and a dedicated medical-information portal. Strategic technology and research partnerships include Veeva Systems for commercial CRM and AI-enabled tools and Genomics Ltd. for underdiagnosis analytics across global biobanks. Customer end-markets are highly fragmented across multiple rare-disease patient populations, with named KOL relationships at institutions such as Murdoch Children's Research Institute and Children's Health Ireland anchoring clinical engagement.

Name

BioMarin Pharmaceutical

Legal name

BioMarin Pharmaceutical Inc.

Website

https://biomarin.com

Company type

Public

Founded year

1997

Operating status

Operating

Headcount range

1,001–5,000 employees

Short description

BioMarin Pharmaceutical is a publicly traded global rare-disease biotechnology company developing and commercializing nine approved therapies for genetically defined conditions, including enzyme replacement, gene therapy, and CNP analog platforms targeting skeletal, lysosomal storage, and metabolic disorders across roughly 80 countries.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Pharmaceutical and Medicine Manufacturing (3254), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

akta.pro secondary industry

Rare Endocrine & Growth Disorder Therapies (HLAIAIAJ)

Keywords

• Rare disease therapeutics
• Enzyme replacement therapies
• Biopharmaceutical products
• Specialty pharmaceuticals
• Gene therapy treatments

Headquarters

HQ city

San Rafael

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Supply Chain, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Commercialized Therapies: BioMarin generates revenue through the sale of FDA-approved rare disease therapeutics including VOXZOGO, enzyme therapies (Vimizim, Naglazyme, Aldurazyme), and acquired products (Galafold, Pombiliti+Opfolda, Palynziq). Revenue grew 13% year-over-year to $3.22 billion in 2025.
2. Royalty Revenue: BioMarin receives royalties from licensed products including Kuvan and Roctavian. However, the company is experiencing headwinds from declining Kuvan royalties and Roctavian withdrawal from the market in 2026.
3. Pipeline Product Revenue: Following the acquisition of Amicus Therapeutics in Q2 2026, BioMarin added GALAFOLD and POMBILITI + OPFOLDA to its commercial portfolio, contributing immediate revenue with expected peak sales of approximately $1 billion.

Pricing tiers

Go-to-market motion3 records

Distribution channels3 records

Marketing channels6 records

Core offering

BioMarin Pharmaceutical develops and commercializes biopharmaceuticals targeting rare genetic and metabolic diseases. The company sells nine commercial therapies including enzyme replacement therapies (Vimizim, Naglazyme, Aldurazyme, Brineura), the CNP analog VOXZOGO for achondroplasia, PKU treatments (Kuvan, Palynziq), and products acquired through the 2026 Amicus Therapeutics transaction (Galafold for Fabry disease, Pombiliti+Opfolda for Pompe disease). Distribution occurs through specialty pharmacies and site of care centers across approximately 80 countries.

Product overview

BioMarin Pharmaceutical is a global rare disease biotechnology company with nine commercial therapies for genetically defined conditions. The company's portfolio includes enzyme replacement therapies (VIMIZIM for MPS IVA, NAGLAZYME for MPS VI, ALDURAZYME for MPS I, BRINEURA for CLN2), small molecule therapies (KUVAN and PALYNZIQ for PKU), and the bone growth therapy VOXZOGO for achondroplasia. The company completed its acquisition of Amicus Therapeutics in April 2026, adding GALAFOLD for Fabry disease and POMBILITI + OPFOLDA for Pompe disease. BioMarin also had ROCTAVIAN for hemophilia A, which was voluntarily withdrawn from the market in early 2026. The pipeline includes BMN 333 (long-acting CNP analog for achondroplasia) and BMN 401 (for ENPP1 deficiency).

Differentiator

Problem solved

Functional benefit

Brands

• PKU & Me: Patient support program for people living with phenylketonuria (PKU), providing resources, recipes, and community connections
• RARE Scholars
• Accelerate Change Together (ACT)

Products and services

• VOXZOGO (vosoritide) A C-type natriuretic peptide (CNP) analog indicated for the treatment of achondroplasia in children aged 2 and older. It works by bypassing the FGFR3 signaling pathway to promote bone growth. VOXZOGO revenue grew 26% to $927 million in 2025 with over 4,500 patient-years of clinical and real-world data generated and more than 3,100 individuals treated.
• PALYNZIQ (pegvaliase-pqpz) An enzyme substitution therapy for phenylketonuria (PKU) that reduces blood phenylalanine concentrations. Approved for adults and adolescents aged 12 and older, based on data from the Phase 3 PEGASUS study demonstrating significant blood phenylalanine reduction versus diet alone.
• KUVAN (sapropterin dihydrochloride) A synthetic form of tetrahydrobiopterin (BH4) for PKU that helps increase enzyme activity to reduce phenylalanine levels in BH4-responsive PKU patients. BioMarin receives royalties from this licensed product.
• VIMIZIM (elosulfase alfa)
• NAGLAZYME (galsulfase)
• ALDURAZYME (laronidase)
• BRINEURA (cerliponase alfa)
• GALAFOLD (migalastat) A small molecule pharmacological chaperone for Fabry disease that selectively and reversibly binds to the misfolded form of the alpha-galactosidase A (GLA) enzyme, increasing its activity. Acquired through the Amicus Therapeutics acquisition completed April 27, 2026.
• POMBILITI + OPFOLDA (cipaglucosidase alfa + avalglucosidase alfa) A two-component therapy for late-onset Pompe disease consisting of POMBILITI (a recombinant human GAA enzyme) and OPFOLDA (an enzyme stabilizer). Acquired through the Amicus Therapeutics acquisition completed April 27, 2026.

Quantifiable outcome

• VOXZOGO achieved 2.33 cm/year improvement in annualized growth velocity in hypochondroplasia Phase 3 trial (CANOPY-HCH-3)
• +3 more outcomes

Named customers4 records

Segments4 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Six partnerships are on record, tiered minor and core.

• Ultragenyx, Alexion, GeneDx, BridgeBiominorGTM or Marketing Partner · 1 April 2026BioMarin co-sponsored Ultragenyx's Rare Bootcamp event in Boston in April 2026, a free multi-day forum helping families of children with rare diseases learn about drug development research and connect with experts. The event featured over 20 sessions covering the spectrum from early science to regulatory approval.
• Genomics Ltd.coreStrategic or Co-development Partner · 12 March 2026Genomics Ltd. announced a partnership with BioMarin Pharmaceutical to advance rare disease drug discovery and development. The collaboration analyzes data from multiple global biobanks to identify geographic regions with acute underdiagnosis rates for lysosomal storage disorders including MPS IVA, MPS VI, and CLN2.
• Murdoch Children's Research InstitutecoreStrategic or Co-development Partner · 12 March 2026BioMarin collaborates with leading academic researchers including Dr. Ravi Savarirayan at Murdoch Children's Research Institute in Melbourne, Australia, who serves as Group Leader of Skeletal Biology and founding member of the Skeletal Dysplasia Management Consortium.
• Dimerix LimitedminorStrategic or Co-development Partner · 20 February 2026Dimerix entered into an exclusive license agreement with Everest Medicines to commercialize DMX-200 for FSGS in Greater China, South Korea, and Southeast Asia. BioMarin acquired U.S. rights to DMX-200 as part of the Amicus Therapeutics acquisition.
• Veeva SystemscoreTechnology or Integration · 1 January 2026Veeva Systems expanded its strategic partnerships with BioMarin and Roche in January 2026, securing multi-year agreements positioning the company as a mission-critical technology provider through its Vault CRM platform and AI-enabled commercial tools. The partnership supports BioMarin's commercial operations and customer engagement capabilities.
• National MPS SocietycoreGTM or Marketing Partner · 1 January 2018BioMarin has supported the National MPS Society since 2018, including the Pathways Program that provides face-to-face guidance from trained professionals at no cost for families following MPS diagnosis. The program offers education, emotional support, and practical resources while connecting families to broader communities.

Scale indicators8 records

Recent moves6 records

Expansion highlights7 records

Direct peers

• Ultragenyx Pharmaceutical: Ultragenyx is a direct peer in rare disease therapeutics, developing enzyme replacement therapies, gene therapies, and biologics for ultra-rare genetic conditions. BioMarin and Ultragenyx co-sponsor the Rare Bootcamp and compete in overlapping skeletal and metabolic rare disease spaces (e.g., achondroplasia, XLH).
• Alexion (AstraZeneca Rare Disease): Alexion is a rare disease leader with commercial enzyme replacement and complement therapies (e.g., Soliris, Strensiq). Both companies sell into lysosomal storage and rare metabolic disease specialists via specialty pharmacy channels, and both co-sponsor rare disease patient advocacy initiatives.
• Genzyme (Sanofi): Genzyme pioneered enzyme replacement therapy for rare lysosomal storage disorders (Cerezyme, Fabrazyme, Aldurazyme), and BioMarin's MPS franchise (Vimizim, Naglazyme, Aldurazyme) directly competes in this category. Both serve the same specialist physician base through similar specialty distribution models.
• Vertex Pharmaceuticals: Vertex is a high-growth rare disease biotech focused on small-molecule therapies for cystic fibrosis, sickle cell disease, and other genetic conditions. Both companies pursue category-defining medicines for genetically defined diseases and have similar commercial specialty pharmacy models.
• BridgeBio Pharma: BridgeBio develops targeted therapies for genetic diseases including achondroplasia (infigratinib), directly competing with BioMarin's VOXZOGO franchise. Both companies target rare genetically defined conditions and co-sponsor the Ultragenyx Rare Bootcamp.
• Sarepta Therapeutics: Sarepta is a gene therapy-focused rare disease company (Elevidys for DMD). BioMarin and Sarepta both operate in ultra-rare genetic disease spaces with complex single-administration or limited-distribution therapies and face similar payer, manufacturing, and launch-execution dynamics.
• Alnylam Pharmaceuticals: Alnylam develops RNAi therapeutics for rare diseases (Onpattro, Givlaari, Oxlumo). Both companies pioneer platform-based approaches to rare genetic conditions, distribute through specialty pharmacies, and target ultra-rare patient populations with high-cost therapies.
• Jazz Pharmaceuticals: Jazz Pharmaceuticals has built a rare disease neurology and oncology franchise via targeted acquisitions. Both companies deploy aggressive M&A strategies to expand rare disease portfolios and rely on specialty distribution channels for commercial execution.

Broad incumbents

• Regeneron Pharmaceuticals: Regeneron is a larger biotech with antibody platform capabilities and rare disease exposure (e.g., evinacumab for HoFH). It overlaps with BioMarin's rare disease focus but operates at broader scale with diversified therapeutic areas including ophthalmology and oncology.
• Takeda Pharmaceutical: Takeda acquired Shire and operates one of the largest rare disease franchises globally, including enzyme replacement therapies for Fabry and Hunter syndrome. It is a broad incumbent in BioMarin's space, offering overlapping capabilities as part of a much larger portfolio.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat7 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/BioMarin
• YouTubeyoutube.com/channel/UC9umRgq3we2eCLBK10-XZWQ/featured
• Facebookfacebook.com/BioMarinOfficial
• LinkedInlinkedin.com/company/biomarin
• Instagraminstagram.com/biomarinpharmaceutical

Revenue estimate

Valuation estimate

Number of profiles

Profiles12 records

Subsidiaries1 record

Funding overview

Funding rounds2 records

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A9 records

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about BioMarin Pharmaceutical