Sponsor pipeline

BioMarin Pharmaceutical clinical trial pipeline

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Data updated daily from ClinicalTrials.gov, last refresh 2026-10-16

What changed in the last 30 days

3 trials changed in the last 30 days: 0 stopped, 0 with notable changes, 3 with minor changes only.

  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.

Active trials by phase

Phase 1
1
Phase 1/2
3
Phase 2
7
Phase 2/3
1
Phase 3
5
Phase 4
1
Not charted: 8 active trials with no phase listed on ClinicalTrials.gov (typically behavioural, device, procedure and observational studies).

Active trials top 25 of 26

NCT IDTitleConditionStatusUpdated
Phase 4
NCT07477691 Immune Modulation During Palynziq® Treatment in Adults (IMPALA) Phenylketonuria Recruiting 2026-06-17
Phase 3
NCT07073014 Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia Hypochondroplasia Enrolling by Invitation 2026-09-09
NCT05270837 Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With… Phenylketonuria (PKU) Active, Not Recruiting 2026-07-21
NCT06224907 Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe… Hemophilia A Active, Not Recruiting 2026-03-19
NCT03424018 An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With… Achondroplasia Active, Not Recruiting 2026-03-13
NCT06455059 Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia Hypochondroplasia Active, Not Recruiting 2026-01-20
Phase 2/3
NCT07441876 Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With… Achondroplasia Recruiting 2026-09-24
Phase 2
NCT06382155 A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature Idiopathic Short Stature Recruiting 2026-05-22
NCT07126262 A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36… Hypochondroplasia Recruiting 2026-03-31
NCT06668805 A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or… Noonan Syndrome Active, Not Recruiting 2026-09-23
NCT07573631 An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in… Duchenne Muscular Dystrophy (DMD) Enrolling by Invitation 2026-07-24
NCT02724228 A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children… Achondroplasia Active, Not Recruiting 2026-03-13
NCT03989947 An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With… Achondroplasia Active, Not Recruiting 2026-03-13
NCT04554940 A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia Achondroplasia Active, Not Recruiting 2026-03-13
Phase 1/2
NCT06280209 A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and… Duchenne Muscular Dystrophy Active, Not Recruiting 2026-06-25
NCT04480567 AAV Gene Therapy Study for Subjects with PKU Phenylketonuria (PKU) Active, Not Recruiting 2024-12-12
NCT05121376 A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema Hereditary Angioedema Active, Not Recruiting 2024-05-16
Phase 1
NCT06738017 Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants Alpha 1-Antitrypsin Deficiency Active, Not Recruiting 2026-05-06
Not specified
NCT06168201 VIrtual STudy in Achondroplasia for the US (VISTA) Achondroplasia Recruiting 2026-06-30
NCT06309979 A Study to Assess Growth in Children With Idiopathic Short Stature Idiopathic Short Stature Recruiting 2026-06-23
NCT05813678 A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) Phenylketonuria (PKU) Recruiting 2026-03-25
NCT05579548 A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to… Phenylketonuria, Maternal Recruiting 2026-03-24
NCT06305234 A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq… Phenylketonuria (PKU) Recruiting 2026-03-24
NCT06212947 A Multicenter Multinational Observational Study of Children With Hypochondroplasia Hypochondroplasia Recruiting 2025-04-03
NCT04476862 Cerliponase Alfa Observational Study in the US Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 Active, Not Recruiting 2026-06-30

Disease areas by active trials

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