BioMarin Pharmaceutical
Open enrollment across a mixed-phase pipeline
According to ClinicalTrials.gov-derived indexes, BioMarin Pharmaceutical sits in the top decile of US sponsors by registered trial count (#280 of 15,742 sponsors by registered trial count).
BioMarin Pharmaceutical: 80 sponsored US clinical trials, 13 recruiting.
BioMarin Pharmaceutical sponsors 80 registered US clinical trials on ClinicalTrials.gov, 13 of them currently recruiting, and 49 completed. 25 of these trials are in Phase 3-4 (later-stage) and 36 in Phase 1-2 (earlier-stage). The most-studied condition in this pipeline is Phenylketonuria (PKU), with 6 trials.
Top-decile sponsor footprint
BioMarin Pharmaceutical ranks in the top decile of sponsors by registered trial count (#280 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.
Trial completion reliability score
49 of 58 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (84.5%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.
Open and recent trials
80 total, page 2 of 2
Extension Study for Patients Who Have Participated in a BMN 701 Study
NCT01435772
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01415427
Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
NCT01387854
Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
NCT01212744
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01275066
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
NCT01230801
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
NCT01114737
Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
NCT00924703
Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU
NCT00925054
Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
NCT00838435
A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
NCT00789568
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
NCT00787995
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT00778206
Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
NCT00634660
A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction
NCT00532844
A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease
NCT00445978
A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT00403494
Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
NCT00355264
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
NCT00332189
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
NCT00299000
A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
NCT00325962
Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
NCT00272792
A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
NCT00225615
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
NCT00214773
Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
NCT00104247
Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
NCT00104260
Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT00104234
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
NCT00067470
Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI
NCT00048711
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
NCT00048620
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 12 |
| Phase 2 | 24 |
| Phase 3 | 22 |
| Phase 4 | 3 |
What is open in the BioMarin Pharmaceutical pipeline
13 recruiting of 80 indexed (16% open); 49 completed (61%).
Phase mix: 25 late (III/IV) vs 36 early (I/II) (balanced across stages).
Top condition: Phenylketonuria (PKU) (6 trials), 9 others listed.
How open-pipeline share is computed
Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.
BioMarin Pharmaceutical: open-pipeline peers nationwide
Peers matched on 80 registered trials and 16% open enrollment, not the therapeutic-area sidebar.
BioMarin Pharmaceutical sits below the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.
Sponsors near 80 trials (± recruiting share)
- Children's Healthcare of Atlanta · 80 trials · 3% recruiting
- EMD Serono Research & Development Institute · 81 trials · 11% recruiting
- The University of Tennessee, Knoxville · 81 trials · 7% recruiting
- TriHealth · 81 trials · 2% recruiting
Sponsors near 16% open enrollment (± trial volume)
- Atlantic Health System · 16% recruiting · 43 trials
- National Human Genome Research Institute (NHGRI) · 16% recruiting · 185 trials
- Drexel University · 16% recruiting · 92 trials
- Case Western Reserve University · 16% recruiting · 141 trials
Related
What to do with this BioMarin Pharmaceutical page
80 registered trials is a research-attention snapshot, not an endorsement of BioMarin Pharmaceutical or any single study.
- 13 of BioMarin Pharmaceutical's trials are currently recruiting, check each one's own eligibility criteria before contacting a site. Browse recruiting trials
- Phenylketonuria (PKU) is the condition BioMarin Pharmaceutical studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Phenylketonuria (PKU) trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
BioMarin Pharmaceutical pipeline card: 80 registered trials from official public datasets.Report a data error.