Total trials
112
Active
27
Phase 3
27
Total enrollment
11,998
Pipeline matrix
Showing 21 of 21 active programs · click any cell for trials
| Intervention | Early Phase 1 | Phase 1 | Phase 2 | Phase 3 | Phase 4 | N/A | Total |
|---|---|---|---|---|---|---|---|
| Pegvaliase | — | — | — | 6 | |||
| Methotrexate | — | — | — | — | — | 1 | |
| RDD to Palynziq | — | — | — | — | — | 1 | |
| Vosoritide | — | — | — | — | 6 | ||
| BMN 111 | — | — | — | 6 | |||
| valoctocogene roxaparvovec | — | — | — | — | 7 | ||
| BMN 333 | — | — | — | — | — | 1 | |
| Vosoritide Injection [Voxzogo] | — | — | — | — | — | 1 | |
| BMN 351 | — | — | — | — | — | 2 | |
| Dose 1 of BMN 331 | — | — | — | — | — | 1 | |
| Dose 2 of BMN 331 | — | — | — | — | — | 1 | |
| Dose 3 of BMN 331 | — | — | — | — | — | 1 | |
| Dose 4 of BMN 331 | — | — | — | — | — | 1 | |
| Dose 5 of BMN 331 | — | — | — | — | — | 1 | |
| Dose 6 of BMN 331 | — | — | — | — | — | 1 | |
| Dose 7 of BMN 331 | — | — | — | — | — | 1 | |
| Human Growth Hormone | — | — | — | — | — | 1 | |
| BMN 307 | — | — | — | — | — | 1 | |
| Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. | — | — | — | — | — | 1 | |
| BMN 349 | — | — | — | — | — | 1 | |
| Cerliponase Alfa | — | — | — | — | — | 1 |
Next readouts
- 2025-01-14
- 2025-04-16
- 2026-06
- 2026-08
- 2026-08-01
- 2026-08-19
- 2027-03
- 2027-04-14
- 2027-09-30
- 2027-12
Top conditions
- Phenylketonuria18
- Achondroplasia14
- Hemophilia A8
- Morquio A Syndrome8
- Duchenne Muscular Dystrophy8
- Phenylketonuria (PKU)7
- Mucopolysaccharidosis IVA6
- MPS IVA6
Frequently asked questions about BioMarin Pharmaceutical
- How many clinical trials does BioMarin Pharmaceutical have on ClinicalTrials.gov?
- BioMarin Pharmaceutical is listed as the lead sponsor on 112 clinical trials in ClinicalTrials.gov, 27 of which are currently active (recruiting, enrolling, or ongoing).
- How many of BioMarin Pharmaceutical's trials are in Phase 3?
- 27 of BioMarin Pharmaceutical's trials are in Phase 3, spread across 21 active drug programs.
- What therapeutic areas does BioMarin Pharmaceutical focus on?
- BioMarin Pharmaceutical's most-studied conditions on ClinicalTrials.gov are Phenylketonuria, Achondroplasia, Hemophilia A, Morquio A Syndrome, Duchenne Muscular Dystrophy.
- When are BioMarin Pharmaceutical's next clinical trial readouts?
- BioMarin Pharmaceutical has 10 upcoming primary-completion readouts tracked here; the soonest is “Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria”, expected around 2025-01-14.