BioMarin Pharmaceutical
NASDAQ:BMRNBiotechSan Rafael, California, United States · Est. 1989 · 1,100 employees
BioMarin Pharmaceutical is a biotechnology company focused on developing and commercializing innovative therapies for rare and serious diseases, particularly rare genetic conditions and metabolic disorders.
8
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
17
Indications↗
Distinct diseases across all programs
100
Clinical Trials↗
All sponsored trials (all statuses)
14
Orange Book Patents
Across 2 drugs with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
8 drugs in development
Phase 34
Phase 24
Phase 11
Preclinical6
By Therapeutic Area
top 4
Rare Disease
536%
Metabolic & Endocrine
321%
Gastroenterology
17%
Hematology
17%
R&D Momentum
59/100
Pipeline Velocity
15/100
Trial Completion Rate
77/100
Pipeline Concentration
27/100
Pipeline Freshness
65/100
Strategic Signals
- ↗Active in Gastroenterology but thin pipeline — potential licensing opportunity
Activity
Recent Activity
Last 14 days
Jul 11, 2026
Completed
A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors
Jul 7, 2026
Recruiting
A Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Jul 1, 2026
Recruiting
VIrtual STudy in Achondroplasia for the US (VISTA)
Jul 1, 2026
Active, not recruiting
Cerliponase Alfa Observational Study
Earlier
Jun 26, 2026TrialA Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy
Jun 24, 2026TrialA Multicenter, Observational Study to Characterize Growth in Children With Idiopathic Short Stature
Mar 31, 2026TrialImmune Modulation During Palynziq® Treatment in Adults (IMPALA)
Mar 16, 2026TrialGENEr8-2: Cerliponase alfa Earlier Intervention
4 more events
Details
Company Info
Company Overview
$13.5B
Mkt Cap
$1.9B
Revenue
—
Cash
CEOJean-Jacques Bienaimé
Founded1989
Employees1,100
HQSan Rafael, California, United States
ExchangeNASDAQ:BMRN
Websitebiomarin.com
Therapeutic Focus
Rare DiseaseCardiovascularGastroenterologyOtherPsychiatryOncologyHematologyNephrologyMetabolic & EndocrineNeurology
Pipeline
Drug Pipeline
Therapeutic Area
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Gastroenterology(1 drug)
—
1
—
—
—
Gastroenterology(1 drug)
Hematology(1 drug)
1
—
1
—
—
Hematology(1 drug)
Metabolic & Endocrine(3 drugs)
—
—
1
2
—
Metabolic & Endocrine(3 drugs)
Rare Disease(5 drugs)
3
—
2
2
—
Rare Disease(5 drugs)
Unknown / Other(4 drugs)
2
—
2
2
—
Unknown / Other(4 drugs)
Regulatory
Regulatory Timeline
20051 event
FDAMay 31
NAGLAZYME — NAGLAZYME
Application BLA125117 (BLA)
20071 event
FDADec 13
Kuvan — KUVAN
Application NDA022181 (NDA)
20131 event
FDADec 19
Kuvan — KUVAN
Application NDA205065 (NDA)
20171 event
FDAApr 27
BRINEURA — BRINEURA
Application BLA761052 (BLA)
20181 event
FDAMay 24
PALYNZIQ — PALYNZIQ
Application BLA761079 (BLA)
20211 event
FDANov 19
Voxzogo — VOXZOGO
Application NDA214938 (NDA)
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)