BioMarin Pharmaceutical Inc.
BMRNNext catalyst
Vosoritide Injection
Achondroplasia in children (full approval)
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Plus 5 more upcoming events in our data (Phase 3 readout, NDA filing, Phase 2 readout).See the timeline
6 upcoming, 1 past
migalastat HCl Phase 3 Est. Readout
Primary completion for migalastat HCl trial (NCT04020055) in Fabry Disease
SourceFDA PDUFA Date Vosoritide (VOXZOGO)
For Achondroplasia in children (full approval). sNDA for full approval. Verified against the company SEC filing on 2026-10-16.
SourceNDA Filing — VOXZOGO
For Achondroplasia in children. The FDA accepted BioMarin's supplemental NDA for full approval of VOXZOGO (vosoritide) in children with achondroplasia, with a PDUFA target action date of February 28, 2027, based on long-term safety and efficacy data including adult height and body proportionality outcomes.
SourceVosoritide Injection Phase 2 Est. Readout
Primary completion for Vosoritide Injection trial (NCT06668805) in Short Stature Homeobox- Containing Gene SHOX Deficiency
SourceBMN 351 Phase 2 Est. Readout
Estimated primary completion for BMN 351 trial (NCT06280209) in Duchenne Muscular Dystrophy. Topline data typically follows completion by weeks to months.
SourceCipaglucosidase alfa Phase 3 Est. Readout (ROSSELLA)
Primary completion for Cipaglucosidase alfa trial (NCT04808505) in Glycogen Storage Disease Type II Infantile Onset
SourceUpcoming FDA decisions
How BMRN's past catalysts played out
Market Overview
Stock performance and key metrics
BioMarin 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).
WebsiteVoxzogo
Vosoritide
Achondroplasia
Roctavian
Valoctocogene Roxaparvovec
Hemophilia A
Valoctocogene roxaparvovec
Hemophilia A With Inhibitor
BMN 165
Phenylketonuria
N-acetylgalactosamine 4-sulfatase
Mucopolysaccharidosis VI
Vosoritide
Hypochondroplasia
Pegvaliase
Phenylketonuria (PKU)
BMN 110 Every Other Week
MPS IV A
Miglustat
Pompe Disease (Late-onset)
Sapropterin Dihydrochloride
Intermittent Claudication
BMN 110
MPS IV A
BMN165
Phenylketonuria (PKU)
Kuvan®
Phenylketonuria
BMN 110 - Weekly
Mucopolysaccharidosis IV A
BMN 333
Achondroplasia
BMN 110 Weekly
MPS IV A
BMN 110 - Every Other Week
Mucopolysaccharidosis IV A
Cipaglucosidase Alfa
Pompe Disease (Late-onset)
INZ-701
Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency
BMN 111
Achondroplasia
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Phenylketonurias
migalastat HCl
Fabry Disease
Azathioprine (Aza)
Mucopolysaccharidosis I
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)
BMN 165 (rAvPAL-PEG)
Phenylketonuria
BMN 190
Jansky-Bielschowsky Disease
Sapropterin Dihydrochloride and Vitamin C
Endothelial Dysfunction
PRO044 SC
Duchenne Muscular Dystrophy
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily
Achondroplasia
Talazoparib Tosylate
Advanced Breast Cancer
sapropterin
Autistic Disorder
6R BH4
Kidney Disease
PRO044 IV
Duchenne Muscular Dystrophy
Cyclosporine A (CsA)
Mucopolysaccharidosis I
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 165 | Phase 3 | Phenylketonuria | - | - |
N-acetylgalactosamine 4-sulfatase | Phase 3 | Mucopolysaccharidosis VI | - | - |
Vosoritide | Phase 3 | Hypochondroplasia | - | - |
Pegvaliase | Phase 3 | Phenylketonuria (PKU) | - | - |
BMN 110 Every Other Week | Phase 3 | MPS IV A | - | - |
Miglustat | Phase 3 | Pompe Disease (Late-onset) | - | Patent 2038 |
Sapropterin Dihydrochloride | Phase 3 | Intermittent Claudication | - | - |
BMN 110 | Phase 3 | MPS IV A | - | - |
BMN165 | Phase 3 | Phenylketonuria (PKU) | - | - |
Kuvan® | Phase 3 | Phenylketonuria | - | - |
BMN 110 - Weekly | Phase 3 | Mucopolysaccharidosis IV A | - | - |
BMN 333 | Phase 3 | Achondroplasia | - | - |
BMN 110 Weekly | Phase 3 | MPS IV A | - | - |
BMN 110 - Every Other Week | Phase 3 | Mucopolysaccharidosis IV A | - | - |
Cipaglucosidase Alfa | Phase 3 | Pompe Disease (Late-onset) | - | - |
INZ-701 | Phase 3 | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | - | - |
BMN 111 | Phase 3 | Achondroplasia | - | - |
sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin | Phase 3 | Phenylketonurias | - | - |
migalastat HCl | Phase 3 | Fabry Disease | - | - |
Azathioprine (Aza) | Phase 2 | Mucopolysaccharidosis I | - | - |
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) | - | - |
BMN 165 (rAvPAL-PEG) | Phase 2 | Phenylketonuria | - | - |
BMN 190 | Phase 2 | Jansky-Bielschowsky Disease | - | - |
Sapropterin Dihydrochloride and Vitamin C | Phase 2 | Endothelial Dysfunction | - | - |
PRO044 SC | Phase 2 | Duchenne Muscular Dystrophy | - | - |
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily | Phase 2 | Achondroplasia | - | - |
Talazoparib Tosylate | Phase 2 | Advanced Breast Cancer | - | - |
sapropterin | Phase 2 | Autistic Disorder | - | - |
6R BH4 | Phase 2 | Kidney Disease | - | - |
PRO044 IV | Phase 2 | Duchenne Muscular Dystrophy | - | - |
Cyclosporine A (CsA) | Phase 2 | Mucopolysaccharidosis I | - | - |
BMN 331 | Phase 1/2 | Phenylketonuria | OrphanFast Track | - |
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
Pompe Disease
1 drug in this indication
late-onset Pompe disease
1 drug in this indication
hypochondroplasia
1 drug in this indication
achondroplasia and hypochondroplasia
1 drug in this indication
Pompe Disease (Late-onset)
1 drug in this indication
Achondroplasia
1 drug in this indication
Source: CatalystAlert database (ClinicalTrials.gov, SEC EDGAR)
Frequently asked questions
What is in BioMarin Pharmaceutical Inc.'s drug pipeline?→
CatalystAlert tracks 43 drug candidates in BioMarin Pharmaceutical Inc.'s (BMRN) pipeline across Phase 3, Phase 2, Phase 1/2, Approved. Its lead program is Vosoritide Injection (Phase 2). 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 date expected Feb 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.