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
32
Phase 3 Programs
14
Upcoming Catalysts
5
Next Catalyst
Aug 3, 2026
5dMarket Overview
Stock performance and key metrics
8 upcoming, 0 past
Placebo Phase 3 Results Expected
Primary completion for Placebo trial (NCT06455059) in Hypochondroplasia
Study met primary endpoint with statistically significant improvement in annualized growth velocity of 2.33 cm/yr compared to placebo at week 52, with significant increases in standing height
SourceVosoritide Phase 3 Results Expected
Primary completion for Vosoritide trial (NCT06455059) in Hypochondroplasia
Phase 3 trial showed efficacy solidly above what the drug demonstrated for achondroplasia
SourceBMRN Earnings Report
EPS Est: $0.79 | FQ end 2026-06
5 more catalysts beyond 14 days
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How BMRN's past catalysts played out
Voxzogo
Vosoritide
Achondroplasia
Roctavian
Valoctocogene Roxaparvovec
Hemophilia A
Valoctocogene roxaparvovec
Hemophilia A With Inhibitor
BMN 111
Achondroplasia
BMN 110
MPS IV A
BMN 165
Phenylketonuria
BMN165 20mg/day
Phenylketonuria (PKU)
N-acetylgalactosamine 4-sulfatase
Mucopolysaccharidosis VI
Vosoritide
Hypochondroplasia
BMN 110 - Weekly
Mucopolysaccharidosis IV A
Pegvaliase
Phenylketonuria (PKU)
Kuvan®
Phenylketonuria
BMN 333
Achondroplasia
Sapropterin Dihydrochloride
Intermittent Claudication
BMN 110 Weekly
MPS IV A
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Phenylketonurias
BMN 351
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
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
PRO044 SC
Duchenne Muscular Dystrophy
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 | - |
Valoctocogene roxaparvovec | Phase 3 | Hemophilia A With Inhibitor | - | - |
BMN 111 | Phase 3 | Achondroplasia | - | - |
BMN 110 | Phase 3 | MPS IV A | - | - |
BMN 165 | Phase 3 | Phenylketonuria | - | - |
BMN165 20mg/day | Phase 3 | Phenylketonuria (PKU) | - | - |
N-acetylgalactosamine 4-sulfatase | Phase 3 | Mucopolysaccharidosis VI | - | - |
Vosoritide | Phase 3 | Hypochondroplasia | - | - |
BMN 110 - Weekly | Phase 3 | Mucopolysaccharidosis IV A | - | - |
Pegvaliase | Phase 3 | Phenylketonuria (PKU) | - | - |
Kuvan® | Phase 3 | Phenylketonuria | - | - |
BMN 333 | Phase 3 | Achondroplasia | - | - |
Sapropterin Dihydrochloride | Phase 3 | Intermittent Claudication | - | - |
BMN 110 Weekly | Phase 3 | MPS IV A | - | - |
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | Phase 3 | Phenylketonurias | - | - |
BMN 351 | 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 | - | - |
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 | - | - |
PRO044 SC | Phase 2 | Duchenne Muscular Dystrophy | - | - |
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)
Full breakdown — the signals behind the verdict
Frequently asked questions
What is in BioMarin Pharmaceutical Inc.'s drug pipeline?→
CatalystAlert tracks 32 drug candidates in BioMarin Pharmaceutical Inc.'s (BMRN) pipeline across Phase 3, Phase 2, Phase 1/2, Approved. Its lead program is Valoctocogene roxaparvovec (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 Earnings expected August 3, 2026. 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.