Biomarin Pharmaceutical Inc. — Sponsor Intelligence | 12 EU Trials

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12 Registered trials

4 Recruiting

6 Completed trials

8 Countries active

5 Unique IMPs

100% Results reported

Phase III

Phase II

Phase III: 4 Phase II/III: 1 Phase II: 5 Phase IV: 1 Phase I (FiH): 1

Oversight events:

1 corrective measure across 1 trial

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100%

6 of 6 completed trials have published summary results (EU CTR Article 37 — 12-month obligation).

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Top indications

Hypochondroplasia 3 Duchenne muscular dystrophy 2 Turner's syndrome 1 Short stature homeobox gene mutation 1 Noonan syndrome 1

Top IMPs

VOSORITIDE 8 BMN 351 2 BMN 333 1 PEGVALIASE 1 VALOCTOCOGENE ROXAPARVOVEC 1

All trials

12 total

An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants with Duchenne Muscular Dystrophy Pending

2024-511656-41-00 Phase II 3 countries BMN 351
A study of BMN 333 versus Vosoritide in Children with Achondroplasia Pending

2025-523811-12-00 Phase II/III 3 countries BMN 333; VOSORITIDE
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and … Recruiting

2024-512261-14-00 Phase II 3 countries VOSORITIDE
Long-Term Extension Study of Vosoritide to Treat Children with Hypochondroplasia Recruiting

2024-517238-16-00 Phase III 4 countries VOSORITIDE
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature Recruiting

2024-520137-74-00 Phase II 3 countries VOSORITIDE
A Basket Study of Vosoritide in Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome with I… Recruiting

2024-515861-33-00 Phase II 4 countries VOSORITIDE
A study to investigate long-term follow-up in subjects who received BMN 270 in a prior BioMarin Clinical Trial with hemophilia A Ended

2023-507749-27-00 Phase IV 5 countries VALOCTOCOGENE ROXAPARVOVEC
A Phase 1/2, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Multiple Intrav… Ended

2023-506737-30-00 Phase I (FiH) 3 countries BMN 351
A Phase 3 Multi-Center Study to Evaluate the Safety and Efficacy of Subcutaneous Injections of Pegvaliase in Adolescent Subjects (Ages 12- 1… Ended

2024-510875-39-00 Phase III 1 country PEGVALIASE
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia Ended

2023-508864-31-00 Phase III 2 countries VOSORITIDE
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children Wit… Ended

2024-513129-22-00 Phase III 4 countries VOSORITIDE
A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia Ended

2023-508754-26-00 Phase II 1 country VOSORITIDE

Showing 1–12 of 12 trials

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