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.
9
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
16
Indications↗
Distinct diseases across all programs
104
Clinical Trials↗
All sponsored trials (all statuses)
14
Orange Book Patents
Across 2 drugs with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
9 drugs in development
Approved1
Phase 36
Phase 24
Preclinical4
By Therapeutic Area
top 4
Rare Disease
427%
Metabolic & Endocrine
213%
Hematology
17%
Neurology
17%
R&D Momentum
61/100
Pipeline Velocity
15/100
Trial Completion Rate
77/100
Pipeline Concentration
32/100
Pipeline Freshness
64/100
Strategic Signals
- ↗Active in Metabolic & Endocrine but thin pipeline — potential licensing opportunity
Activity
Recent Activity
Earlier
Aug 25, 2026TrialA Phase 2, Randomized, Multicenter, Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Aug 22, 2026TrialA Multicenter, Randomized, Operationally Seamless Phase 2/3 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Jul 25, 2026TrialAn Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of Weekly Intravenous Infusions of BMN 351 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Jul 22, 2026TrialA Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12-17) With Phenylketonuria- Open-Label Randomized Two-Arm (Active vs Diet-Only Control)
Jul 11, 2026TrialA 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 1, 2026TrialVIrtual STudy in Achondroplasia for the US (VISTA)
Jul 1, 2026TrialCerliponase Alfa Observational Study
4 more events
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
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 DiseaseMetabolic & EndocrineOtherNeurologyPsychiatryGastroenterologyCardiovascularHematologyRespiratoryOncology
Pipeline
Drug Pipeline
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Rare Disease
Hematology
Metabolic & Endocrine
Turner Syndrome
VoxzogoPhase 2
Neurology
Rare Disease
Unknown / Other
Disease Progression
BRINEURAPhase 2
Gene Therapy
BMN 333Phase 2
Hypochondroplasia
VoxzogoPhase 3
Noonan Syndrome
VoxzogoPhase 2
Short Stature
VoxzogoPhase 2
Treatment
PegvaliaseApproved
Showing 15 of 19 programs
Regulatory
Regulatory Timeline
20071 event
FDADec 13
Kuvan — KUVAN
Application NDA022181 (NDA)
20132 events
FDADec 19
Kuvan — KUVAN
Application NDA205065 (NDA)
FDADec 19
Kuvan — KUVAN
Application NDA205065 (NDA)
20172 events
FDAApr 27
BRINEURA — BRINEURA
Application BLA761052
FDAApr 27
BRINEURA — BRINEURA
Application BLA761052 (BLA)
20183 events
FDADec 11
BRINEURA — BRINEURA
Application BLA761052
FDAMay 24
PALYNZIQ — PALYNZIQ
Application BLA761079 (BLA)
FDAMay 24
Pegvaliase — PALYNZIQ
Application BLA761079
20191 event
FDADec 6
BRINEURA — BRINEURA
Application BLA761052
20205 events
FDANov 20
Pegvaliase — PALYNZIQ
Application BLA761079
FDAOct 6
Pegvaliase — PALYNZIQ
Application BLA761079
FDAOct 6
Pegvaliase — PALYNZIQ
Application BLA761079
FDAOct 6
Pegvaliase — PALYNZIQ
Application BLA761079
FDAMar 27
BRINEURA — BRINEURA
Application BLA761052
20211 event
FDANov 19
Voxzogo — VOXZOGO
Application NDA214938 (NDA)
20231 event
FDADec 9
Pegvaliase — PALYNZIQ
Application BLA761079
20242 events
FDAJul 24
BRINEURA — BRINEURA
Application BLA761052
FDAJul 10
BRINEURA — BRINEURA
Application BLA761052
20251 event
FDANov 20
Pegvaliase — PALYNZIQ
Application BLA761079
20261 event
FDAFeb 27
Pegvaliase — PALYNZIQ
Application BLA761079
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)