BioMarin Pharmaceutical Inc.
BMRNBioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research are in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing laronidase (Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first company to provide therapeutics for phenylketonuria (PKU).
Drugs in Pipeline
33
Phase 3 Programs
15
Upcoming Catalysts
3
Next Catalyst
Aug 3, 2026
7wMarket Overview
Stock performance and key metrics
8 upcoming, 0 past
Vosoritide Injection Phase 2 Results Expected
Primary completion for Vosoritide Injection trial (NCT06382155) in Idiopathic Short Stature
Phase 2 study discontinuations
SourcePlacebo Phase 2 Results Expected
Primary completion for Placebo trial (NCT06382155) in Idiopathic Short Stature
BioMarin discontinues dosing in Phase II trials due to safety concern
SourceHuman Growth Hormone Phase 2 Results Expected
Primary completion for Human Growth Hormone trial (NCT06382155) in Idiopathic Short Stature
BioMarin discontinuing dosing and enrollment in Phase 2 Voxzogo studies
Source5 more catalysts beyond 14 days
Start a 7-day free trial to see all upcoming catalysts
Roctavian
Valoctocogene Roxaparvovec
Hemophilia A
Voxzogo
Vosoritide
Achondroplasia
Roxadustat
Anemia Associated With End Stage Renal Disease
N1539
Pain, Post-operative
BMN 111
Achondroplasia
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Phenylketonurias
Valoctocogene roxaparvovec
Hemophilia A With Inhibitor
BMN 165
Phenylketonuria
N-acetylgalactosamine 4-sulfatase
Mucopolysaccharidosis VI
Vosoritide
Hypochondroplasia
BMN 110
MPS IV A
Pegvaliase
Phenylketonuria (PKU)
BMN 110 - Weekly
Mucopolysaccharidosis IV A
Kuvan®
Phenylketonuria
Sapropterin Dihydrochloride
Intermittent Claudication
BMN165 20mg/day
Phenylketonuria (PKU)
BMN 110 Weekly
MPS IV A
PRO044 SC
Duchenne Muscular Dystrophy
Human Growth Hormone
Idiopathic Short Stature
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
Jansky-Bielschowsky Disease
Vosoritide Injection
Short Stature Homeobox- Containing Gene SHOX Deficiency
rAvPAL-PEG
Phenylketonuria
Phenoptin
Tetrahydrobiopterin Deficiencies
BMN 701
Pompe Disease
BMN 351
Duchenne Muscular Dystrophy
6R-BH4 (sapropterin dihydrochloride)
Hypertension
BMN 307
Phenylketonuria (PKU)
rAvPAL-PEG 0.001 mg/kg
Phenylketonuria
BMN 165 (rAvPAL-PEG)
Phenylketonuria
BMN 190
Jansky-Bielschowsky Disease
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Achondroplasia
Sapropterin Dihydrochloride and Vitamin C
Endothelial Dysfunction
BMN 331
Phenylketonuria
| Drug Name | Phase | Indication | Designations | Patent |
|---|---|---|---|---|
Roctavian Valoctocogene Roxaparvovec | Approved | Hemophilia A | OrphanFast TrackBreakthrough | - |
Voxzogo Vosoritide | Approved | Achondroplasia | OrphanFast TrackBreakthrough | Patent 2042 |
Roxadustat | Phase 3 | Anemia Associated With End Stage Renal Disease | - | - |
N1539 | Phase 3 | Pain, Post-operative | - | - |
BMN 111 | Phase 3 | Achondroplasia | - | - |
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | Phase 3 | Phenylketonurias | - | - |
Valoctocogene roxaparvovec | Phase 3 | Hemophilia A With Inhibitor | - | - |
BMN 165 | Phase 3 | Phenylketonuria | - | - |
N-acetylgalactosamine 4-sulfatase | Phase 3 | Mucopolysaccharidosis VI | - | - |
Vosoritide | Phase 3 | Hypochondroplasia | - | - |
BMN 110 | Phase 3 | MPS IV A | - | - |
Pegvaliase | Phase 3 | Phenylketonuria (PKU) | - | - |
BMN 110 - Weekly | Phase 3 | Mucopolysaccharidosis IV A | - | - |
Kuvan® | Phase 3 | Phenylketonuria | - | - |
Sapropterin Dihydrochloride | Phase 3 | Intermittent Claudication | - | - |
BMN165 20mg/day | Phase 3 | Phenylketonuria (PKU) | - | - |
BMN 110 Weekly | Phase 3 | MPS IV A | - | - |
PRO044 SC | Phase 2 | Duchenne Muscular Dystrophy | - | - |
Human Growth Hormone | Phase 2 | Idiopathic Short Stature | - | - |
BMN 190 recombinant human tripeptidyl peptidase-1 (rhTPP1) | Phase 2 | Jansky-Bielschowsky Disease | - | - |
Vosoritide Injection | Phase 2 | Short Stature Homeobox- Containing Gene SHOX Deficiency | - | - |
rAvPAL-PEG | Phase 2 | Phenylketonuria | - | - |
Phenoptin | Phase 2 | Tetrahydrobiopterin Deficiencies | - | - |
BMN 701 | Phase 2 | Pompe Disease | - | - |
BMN 351 | Phase 2 | Duchenne Muscular Dystrophy | - | - |
6R-BH4 (sapropterin dihydrochloride) | Phase 2 | Hypertension | - | - |
BMN 307 | Phase 2 | Phenylketonuria (PKU) | - | - |
rAvPAL-PEG 0.001 mg/kg | Phase 2 | Phenylketonuria | - | - |
BMN 165 (rAvPAL-PEG) | Phase 2 | Phenylketonuria | - | - |
BMN 190 | Phase 2 | Jansky-Bielschowsky Disease | - | - |
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. | Phase 2 | Achondroplasia | - | - |
Sapropterin Dihydrochloride and Vitamin C | Phase 2 | Endothelial Dysfunction | - | - |
BMN 331 | Phase 1/2 | Phenylketonuria | OrphanFast Track | - |
Achondroplasia
1 drug in this indication
Hemophilia A
1 drug in this indication
Phenylketonuria
1 drug in this indication
Source: CatalystAlert database (ClinicalTrials.gov, SEC EDGAR)