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
36
Phase 3 Programs
15
Upcoming Catalysts
4
Next Catalyst
Feb 28, 2027
24wMarket Overview
Stock performance and key metrics
6 upcoming, 1 past
How BMRN's past catalysts played out
Voxzogo
Vosoritide
Achondroplasia
Roctavian
Valoctocogene Roxaparvovec
Hemophilia A
BMN 165
Phenylketonuria
BMN 333
Achondroplasia
BMN 110 - Weekly
Mucopolysaccharidosis IV A
N-acetylgalactosamine 4-sulfatase
Mucopolysaccharidosis VI
Vosoritide
Hypochondroplasia
Kuvan®
Phenylketonuria
Pegvaliase
Phenylketonuria (PKU)
INZ-701
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
BMN 110 Weekly
MPS IV A
Sapropterin Dihydrochloride
Intermittent Claudication
BMN 110
MPS IV A
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Phenylketonurias
BMN165 20mg/day
Phenylketonuria (PKU)
Valoctocogene roxaparvovec
Hemophilia A With Inhibitor
BMN 111
Achondroplasia
PRO044 SC
Duchenne Muscular Dystrophy
6R BH4
Kidney Disease
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
Talazoparib Tosylate
Advanced Breast Cancer
sapropterin
Autistic Disorder
BMN 331
Phenylketonuria
| Drug Name | Phase | Indication | Designations | Patent |
|---|---|---|---|---|
Voxzogo Vosoritide | Approved | Achondroplasia | OrphanFast TrackBreakthrough | Patent 2042 |
Roctavian Valoctocogene Roxaparvovec | Approved | Hemophilia A | OrphanFast TrackBreakthrough | - |
BMN 165 | Phase 3 | Phenylketonuria | - | - |
BMN 333 | Phase 3 | Achondroplasia | - | - |
BMN 110 - Weekly | Phase 3 | Mucopolysaccharidosis IV A | - | - |
N-acetylgalactosamine 4-sulfatase | Phase 3 | Mucopolysaccharidosis VI | - | - |
Vosoritide | Phase 3 | Hypochondroplasia | - | - |
Kuvan® | Phase 3 | Phenylketonuria | - | - |
Pegvaliase | Phase 3 | Phenylketonuria (PKU) | - | - |
INZ-701 | Phase 3 | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | - | - |
BMN 110 Weekly | Phase 3 | MPS IV A | - | - |
Sapropterin Dihydrochloride | Phase 3 | Intermittent Claudication | - | - |
BMN 110 | Phase 3 | MPS IV A | - | - |
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | Phase 3 | Phenylketonurias | - | - |
BMN165 20mg/day | Phase 3 | Phenylketonuria (PKU) | - | - |
Valoctocogene roxaparvovec | Phase 3 | Hemophilia A With Inhibitor | - | - |
BMN 111 | Phase 3 | Achondroplasia | - | - |
PRO044 SC | Phase 2 | Duchenne Muscular Dystrophy | - | - |
6R BH4 | Phase 2 | Kidney Disease | - | - |
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 | - | - |
Talazoparib Tosylate | Phase 2 | Advanced Breast Cancer | - | - |
sapropterin | Phase 2 | Autistic Disorder | - | - |
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
ENPP1 deficiency
1 drug in this indication
rare disease
1 drug in this indication
Source: CatalystAlert database (ClinicalTrials.gov, SEC EDGAR)
Full breakdown — the signals behind the verdict
Frequently asked questions
What is in BioMarin Pharmaceutical Inc.'s drug pipeline?→
CatalystAlert tracks 36 drug candidates in BioMarin Pharmaceutical Inc.'s (BMRN) pipeline across Phase 3, Phase 2, Phase 1/2, Approved. Its lead program is BMN 165 (Phase 3). Each drug's phase, indication and upcoming catalysts are listed on this page.
When is BioMarin Pharmaceutical Inc.'s next FDA catalyst?→
BioMarin Pharmaceutical Inc.'s (BMRN) next tracked catalyst is a PDUFA expected February 28, 2027. See the catalyst timeline on this page for the full schedule of PDUFA dates, trial readouts and FDA events.
How do I track BMRN biotech catalysts?→
Add BMRN to your CatalystAlert watchlist for alerts ahead of each catalyst, and follow BioMarin Pharmaceutical Inc.'s live catalyst calendar, drug pipeline and clinical-trial timeline on this page — sourced from ClinicalTrials.gov, FDA and SEC filings.