BioMarin Pharmaceutical Inc (BMRN) — Upcoming Catalysts

FDA PDUFA dates, advisory-committee meetings, and Phase 2/3 readout windows for BioMarin Pharmaceutical Inc, built from public data lanes (ClinicalTrials.gov and official sources). Each row links to its source.

For informational purposes only — not investment advice. Track BMRN live on Mooboard →

Later12

  • Mar 2027
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    Vosoritide Injection · Phase 2 · Noonan Syndrome
    NCT06668805: A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment. Primary completion 2027-03 (estimated); status RECRUITING.
    ClinicalTrials.gov ↗
  • Apr 14, 2027
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    BMN 351 · Phase 1 / Phase 2 · Duchenne Muscular Dystrophy
    NCT06280209: A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy. Primary completion 2027-04-14 (estimated); status ACTIVE_NOT_RECRUITING.
    ClinicalTrials.gov ↗
  • Dec 2027
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    BMN 111 · Phase 2 · Achondroplasia
    NCT02724228: A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH). Primary completion 2027-12 (estimated); status ACTIVE_NOT_RECRUITING.
    ClinicalTrials.gov ↗
  • Dec 2027
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    vosoritide · Phase 2 · Achondroplasia
    NCT04554940: A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia. Primary completion 2027-12 (estimated); status ACTIVE_NOT_RECRUITING.
    ClinicalTrials.gov ↗
  • Dec 2027
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    BMN 307 · Phase 1 / Phase 2 · Phenylketonuria (PKU)
    NCT04480567: AAV Gene Therapy Study for Subjects with PKU. Primary completion 2027-12 (estimated); status ACTIVE_NOT_RECRUITING.
    ClinicalTrials.gov ↗
  • Jun 30, 2028
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    Vosoritide · Phase 2 · Hypochondroplasia
    NCT07126262: A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months. Primary completion 2028-06-30 (estimated); status RECRUITING.
    ClinicalTrials.gov ↗
  • Nov 2028
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    Dose 1 of BMN 331 · Phase 1 / Phase 2 · Hereditary Angioedema; HAE
    NCT05121376: A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema. Primary completion 2028-11 (estimated); status ACTIVE_NOT_RECRUITING.
    ClinicalTrials.gov ↗
  • Jun 2029
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    BMN 333 · Phase 2 / Phase 3 · Achondroplasia
    NCT07441876: Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia. Primary completion 2029-06 (estimated); status RECRUITING.
    ClinicalTrials.gov ↗
  • Jun 2031
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    BMN 111 · Phase 3 · Achondroplasia
    NCT03424018: An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia. Primary completion 2031-06 (estimated); status ACTIVE_NOT_RECRUITING.
    ClinicalTrials.gov ↗
  • Dec 2031
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    BMN 351 · Phase 2 · Duchenne Muscular Dystrophy (DMD)
    NCT07573631: An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy. Primary completion 2031-12 (estimated); status ENROLLING_BY_INVITATION.
    ClinicalTrials.gov ↗
  • May 2038
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. · Phase 2 · Achondroplasia
    NCT03989947: An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia. Primary completion 2038-05 (estimated); status ACTIVE_NOT_RECRUITING.
    ClinicalTrials.gov ↗
  • Dec 2040
    BioMarin Pharmaceutical Inc(BMRN)Phase 2/3 readout
    Vosoritide · Phase 3 · Hypochondroplasia
    NCT07073014: Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia. Primary completion 2040-12 (estimated); status ENROLLING_BY_INVITATION.
    ClinicalTrials.gov ↗

How BioMarin Pharmaceutical Inc has moved — past-year price reactions

How binary is BMRN? Its typical daily move, how often it swings ±10%, and its biggest single-day moves over the last year — useful context for sizing around a catalyst.

1.1%
typical day (1Y)
1
±10% days
34.2%
annualized vol
250
trading days
Largest single-day moves
  • +17.7%2025-12-19no matched headline
  • +8.6%2026-05-29no matched headline
  • +7.8%2026-05-21no matched headline
  • -4.3%2026-01-13no matched headline
  • +4.3%2025-11-11no matched headline
  • +4.2%2025-11-21no matched headline

Real daily price moves over the past year — not every move is a catalyst, and headlines (where shown) are a possible same-day driver, not confirmed causation.

BioMarin Pharmaceutical Inc — active clinical pipeline

BioMarin Pharmaceutical Inc's registered Phase 1–3 programs by asset and indication, derived from ClinicalTrials.gov.

12 assets18 programsPh1 1Ph2 10Ph3 5· 2 post-marketing (Ph4)
AreasRare / GeneticOther
VosoritideOther · NPR2
Phase 2
AchondroplasiaHypochondroplasia
Phase 3
Hypochondroplasia (2)
BMN 111Other · NPR2
Phase 2
Achondroplasia
Phase 3
Achondroplasia
BMN 333Other
Phase 3
Achondroplasia
PegvaliaseOther · Phenylalanine
Phase 3
Phenylketonuria (PKU)
Valoctocogene roxaparvovecRare / Genetic · Factor IX
Phase 3
Hemophilia A
BMN 351Rare / Genetic
Phase 2
Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)
Vosoritide InjectionOther
Phase 2
Idiopathic Short StatureNoonan Syndrome
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.Other
Phase 2
Achondroplasia
BMN 307Other
Phase 2
Phenylketonuria (PKU)
Dose 1 of BMN 331Rare / Genetic
Phase 2
Hereditary Angioedema
BMN 349Other
Phase 1
Alpha 1-Antitrypsin Deficiency

Active registered Phase 1–3 trials from ClinicalTrials.gov — not the company's official IR pipeline (no preclinical or undisclosed assets). Grouped by trial intervention name; therapeutic area is auto-categorized. Each indication links to its trial. Molecular targets sourced from ChEMBL (EMBL-EBI).

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