Nasdaq · Biotech stocks database
BMRN — BioMarin Pharmaceutical Inc.
BMRN (BioMarin Pharmaceutical Inc.) stock: $63.94, $12.38B market cap, $874.0M cash, $15.03B EV, $13.41B fully diluted market cap. Next expected readout: Mar-27. Lead program: BMN 333 (Achondroplasia), Phase 3. Data from Fully Diluted's biotech stocks database.
Overview
Assets
Pipeline
Trials
Holders
Enterprise value
$15,031.0M
Balance sheet
Cash $874.0M Debt $3,527.9M Net cash -$2,653.9M Enterprise value $15,031.0M Basic shares 193.5M shares Fully diluted shares 209.8M shares Fully diluted market cap $13,414.0M Cash per share $4.52
Cash figures as of 2026-06-30. Database snapshot: 2026-09-22.
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Upcoming catalysts Expected Drug Phase Indication Mgmt guide Now - Oct 24, 2026 Vosoritide Phase 3 Hypochondroplasia — Sep 30 - Nov 11 BMN 349 Phase 1 Alpha 1-Antitrypsin Deficiency — Nov 25, 2026 - Jan 6, 2027 INZ-701 Phase 3 Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency; Autosomal Recessive Hypophosphatemic Rickets; Generalized Arterial Calcification of Infancy 1 — Nov 25, 2026 - Jan 6, 2027 INZ-701 Phase 1 Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency; Autosomal Recessive Hypophosphatemic Rickets; Generalized Arterial Calcification of Infancy — Apr 12 - May 24 Vosoritide Injection Phase 2 Noonan Syndrome — May 26 - Jul 7 BMN 351 Phase 2 Duchenne Muscular Dystrophy — Jan 12 - Feb 23 BMN 111 Phase 2 Achondroplasia — Jan 12 - Feb 23 BMN 307 Phase 2 Phenylketonuria (PKU) — Jan 12 - Feb 23 vosoritide Phase 2 Achondroplasia — Aug 11 - Sep 22 Vosoritide Phase 2 Hypochondroplasia — Jul 13 - Aug 24 BMN 333 Phase 3 Achondroplasia 2027 Jul 13 - Aug 24 BMN 111 Phase 3 Achondroplasia — Jan 12 - Feb 23 BMN 351 Phase 2 Duchenne Muscular Dystrophy (DMD) — Nov 12 - Dec 24 Vosoritide Phase 3 Hypochondroplasia —
Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Full readout calendar →
Pipeline assets VOXZOGO
Approved · Active
Aliases vosoritide
Target FGFR3 pathway
Indications Achondroplasia (Approved) Hypochondroplasia (Phase 3) Idiopathic short stature (Stage not disclosed) Noonan syndrome (Stage not disclosed) Status note Phase 3 CANOPY-HCH-3 study in hypochondroplasia met its primary endpoint (May 2026); sNDA submitted July 2026.
PALYNZIQ
Approved · Active
Aliases pegvaliase-pqpz
Target phenylalanine
Indications Phenylketonuria (PKU) (Approved) Status note European Commission approved for adolescents 12+ (Jun 2026); FDA approved for adolescents 12+ (Feb 2026).
BRINEURA
Approved · Active
Aliases cerliponase alfa
Target TPP1
Indications CLN2 disease (Batten disease) (Approved) Status note Approved commercial product.
NAGLAZYME
Approved · Active
Aliases galsulfase
Target glycosaminoglycans (GAGs)
Indications Mucopolysaccharidosis VI (MPS VI) (Approved) Status note Approved commercial product.
KUVAN
Approved · Active
Aliases sapropterin dihydrochloride
Target PAH
Indications Phenylketonuria (PKU) (Approved) Status note Generic versions available in the U.S. and EU, adversely affecting revenues.
ALDURAZYME
Approved · Active
Aliases laronidase
Target glycosaminoglycans (GAGs)
Partnerships Marketed worldwide by Sanofi
Indications Mucopolysaccharidosis I (MPS I) (Approved) Status note Marketed worldwide by Sanofi.
GALAFOLD
Approved · Active
Aliases migalastat
Status note First oral treatment for Fabry disease; added via Amicus acquisition (closed April 27, 2026).
POMBILITI + OPFOLDA
Approved · Active
Aliases cipaglucosidase alfa-atga, miglustat
Status note Two-component therapy added via Amicus acquisition (closed April 27, 2026).
BMN 820
Phase 3 · Active
Aliases DMX-200
Target CCR2
Partnerships Dimerix: U.S. commercialization rights licensed from Dimerix; up to $510M milestones plus tiered royalties low-teens to low-twenties
Indications Focal segmental glomerulosclerosis (FSGS) (Phase 3) Status note Potential first-in-class oral CCR2 inhibitor for a rare fatal kidney disease; U.S. rights acquired via Amicus acquisition.
BMN 333
Phase 2/3 · Active Modality: long-acting C-type natriuretic peptide (CNP)
Target CNP
Indications Achondroplasia (Phase 2/3) Status note Registration-enabling Phase 2/3 study; first patient enrolled April 2026.
ROCTAVIAN
Approved · Terminated
Target F8 (factor VIII)
Indications Severe hemophilia A (Approved) Status note In 2026 the company announced it will no longer market ROCTAVIAN.
BMN 401
Phase 3 · Terminated Modality: enzyme replacement therapy
Aliases INZ-701
Target ENPP1
Indications ENPP1 deficiency (Phase 3) Status note Did not meet one of two co-primary endpoints in the pivotal ENERGY 3 trial; development discontinued across all indications August 2026. Acquired via Inozyme Pharma (Jul 2025).
Per-asset detail extracted from the 10-Q filed 2026-08-06. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Pipeline Lead drug BMN 333 Lead indication Achondroplasia Lead phase Phase 3 Active trials 27 Collaborator trials 7
Active clinical trials Sponsor-matched active trials from ClinicalTrials.gov (27 as sponsor, 7 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
View all 34 active trials for BioMarin Pharmaceutical on ClinicalTrials.gov
Institutional holders Fund Shares Q/Q change % of portfolio Filing Sphera Biotech 328,550 +188,650 2.69% 2026-08-14 ADAR1 Capital Management 49,000 +30,800 0.13% 2026-08-14 Braidwell LP 0 -497,020 0.00% 2026-08-14
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
Fully Diluted · Free biotech stocks database. Figures are sourced from SEC filings and company disclosures; not investment advice.