Companies / BioMarin Pharmaceutical
BioMarin Pharmaceutical Pipeline
CompanyBioMarin Pharmaceutical is an American biotechnology company headquartered in San Rafael, California. It focuses on therapies for rare and serious diseases and is publicly traded on Nasdaq as BMRN.
Continuously tracked·last revised
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Recruiting Phase 3
| Trial | Phase | Status | Sponsor | Started |
|---|---|---|---|---|
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia NCT07441876 | Phase 2/3 | Recruiting | BioMarin Pharmaceutical | Started 2026-04 |
| A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months NCT07126262 | Phase 2 | Recruiting | BioMarin Pharmaceutical | Started 2025-07 |
| A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature NCT06382155 | Phase 2 | Recruiting | BioMarin Pharmaceutical | Started 2024-10 |
Recently started
| Trial | Phase | Status | Sponsor | Started |
|---|---|---|---|---|
| Immune Modulation During Palynziq® Treatment in Adults (IMPALA) NCT07477691 | Phase 4 | Recruiting | BioMarin Pharmaceutical | Started 2026-05 |
| An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy NCT07573631 | Phase 2 | Enrolling by invitation | BioMarin Pharmaceutical | Started 2026-04 |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia NCT07441876 | Phase 2/3 | Recruiting | BioMarin Pharmaceutical | Started 2026-04 |
Results recently posted
| Trial | Phase | Status | Sponsor | Results posted |
|---|---|---|---|---|
| Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria NCT05270837 | Phase 3 | Active, not recruiting | BioMarin Pharmaceutical | Results posted 2026-07-21 |
| Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 NCT03520712 | Phase 1/2 | Terminated | BioMarin Pharmaceutical | Results posted 2025-08-22 |
| Gene Therapy Study in Severe Haemophilia A Patients (270-201) NCT02576795 | Phase 1/2 | Completed | BioMarin Pharmaceutical | Results posted 2025-04-10 |
| A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease NCT02678689 | Phase 2 | Completed | BioMarin Pharmaceutical | Results posted 2025-02-17 |
| A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia NCT03583697 | Phase 2 | Completed | BioMarin Pharmaceutical | Results posted 2024-06-13 |
Pipeline
Phase by status
| Phase | Recruiting | By invitation | Active | Completed | Terminated | Withdrawn | Unknown | Approved for marketing | No longer available | Total |
|---|---|---|---|---|---|---|---|---|---|---|
| Phase 1 | — | — | 1 | 4 | 1 | 1 | — | — | — | 7 |
| Phase 1/2 | — | — | 3 | 7 | 4 | — | — | — | — | 14 |
| Phase 2 | 2 | 1 | 4 | 15 | 7 | — | — | — | — | 29 |
| Phase 2/3 | 1 | — | — | — | — | — | — | — | — | 1 |
| Phase 3 | — | 1 | 4 | 19 | 2 | — | — | — | — | 26 |
| Phase 4 | 1 | — | 1 | 2 | — | — | — | — | — | 4 |
| Not applicable | — | — | — | — | 1 | — | — | — | — | 1 |
| Unknown | 6 | 1 | 1 | 13 | 4 | — | 1 | 3 | 1 | 30 |
Trials started per year
Conditions studied, by phase
| Condition | Phase 1 | Phase 1/2 | Phase 2 | Phase 2/3 | Phase 3 | Phase 4 | Not applicable | Unknown | Total |
|---|---|---|---|---|---|---|---|---|---|
| Phenylketonurias | 2 | 1 | 9 | — | 11 | 3 | — | 9 | 35 |
| Achondroplasia | 2 | — | 5 | 1 | 2 | — | — | 4 | 14 |
| Genetic Diseases, Inborn | — | 3 | 1 | 1 | 7 | — | — | 1 | 13 |
| Mucopolysaccharidosis IV | — | 2 | 3 | — | 3 | — | — | 3 | 11 |
| Bone Diseases | — | — | 6 | 1 | 3 | — | — | — | 10 |
| Dwarfism | — | — | 6 | — | 3 | — | — | 1 | 10 |
| Hemophilia A | — | 3 | — | — | 4 | — | 1 | 2 | 10 |
| Muscular Dystrophy, Duchenne | — | 4 | 3 | — | 1 | — | — | 2 | 10 |
| Lysosomal Storage Diseases | — | 2 | 3 | — | 2 | — | — | 2 | 9 |
| Blood Coagulation Disorders | — | 3 | — | — | 4 | — | — | 1 | 8 |
Most active sponsors
| # | Sponsor | Trials | Phase 3 |
|---|---|---|---|
| 1 | BioMarin Pharmaceutical | 112 | 27 |
Frequent collaborators
| # | Collaborator | Trials |
|---|---|---|
| 1 | ICON Clinical Research | 1 |
| 2 | ICON plc | 1 |
| 3 | PicnicHealth | 1 |
| 4 | Quintiles, Inc. | 1 |
| 5 | Syneos Health | 1 |
Most studied interventions
| # | Intervention | Trials |
|---|---|---|
| 1 | sapropterin | 18 |
| 2 | vosoritide | 14 |
| 3 | pegvaliase | 13 |
| 4 | GALNS protein, human | 9 |
| 5 | Valoctocogene Roxaparvovec | 7 |
| 6 | cerliponase alfa | 4 |
| 7 | Chondroitinases and Chondroitin Lyases | 4 |
| 8 | Enzyme Replacement Therapy | 4 |
| 9 | Galns protein, mouse | 4 |
| 10 | N-Acetylgalactosamine-4-Sulfatase | 4 |
Terminations
Stated reasons
Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.
Recently stopped
| Trial | Status | Stated reason | Stopped |
|---|---|---|---|
| NCT03520712 Terminated | Terminated | Early rollover into long-term extension study | 2024-08-07 |
| NCT05580692 Terminated | Terminated | Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved. | 2024-07-31 |
| NCT06138327 Withdrawn | Withdrawn | The study was withdrawn because BioMarin decided to end the overall development program. The study withdrawal was not due to any patient safety concerns. | 2024-03-25 |
| NCT05813314 Terminated | Terminated | The BMN 111-104 study has terminated as further optimization of the approach to achieve bio equivalence is required. The study termination was not due to any patient safety concerns. | 2023-06-14 |
| NCT01753804 Terminated | Terminated | 2016-10-01 | |
| NCT01803412 Terminated | Terminated | 2016-10-01 | |
| NCT01910649 Terminated | Terminated | Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD. | 2016-09-30 |
| NCT02221362 Terminated | Terminated | 2016-09-30 | |
| NCT02958202 Terminated | Terminated | Regulatory approval was not obtained for drisapersen, hence BioMarin is stopping the development of all exon skipping oligonucleotides in DMD. | 2016-09-30 |
| NCT01924845 Terminated | Terminated | Sponsor decision | 2016-09-12 |
Evidence
- Results reported
- 76.7%46 of 60 completed
- Published
- 1.7%1 of 60 completed
- Primary endpoint met
- 84%21 of 25 analyses
- Median months to report
- 21
Documents disclosed
Trial profile
- Median enrollment
- 40
- Median duration
- 41.4months
- Countries
- 44
- Study sites
- 1019
Who is enrolled
Median enrollment by phase
- Phase 1
- 31
- Phase 1/2
- 19
- Phase 2
- 28
- Phase 2/3
- 160
- Phase 3
- 85
- Phase 4
- 11
- Not applicable
- 321
- Unknown
- 186
Where trials run
Busiest cities
| # | City | Country | Trials |
|---|---|---|---|
| 1 | Chicago | United States | 46 |
| 2 | London | United Kingdom | 42 |
| 3 | Oakland | United States | 29 |
| 4 | Houston | United States | 23 |
| 5 | Minneapolis | United States | 23 |
| 6 | Los Angeles | United States | 22 |
| 7 | Milwaukee | United States | 22 |
| 8 | New York | United States | 22 |
| 9 | Portland | United States | 22 |
| 10 | Pittsburgh | United States | 21 |