BioMarin Pharmaceutical
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.
Trial Pipeline
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 the Pipeline for BioMarin Pharmaceutical Shows
According to the ClinicalTrials.gov registry, BioMarin Pharmaceutical is linked to 80 US clinical trials across every stage of research activity. Of those, 13 studies are currently recruiting, about 16% of the sponsor's indexed portfolio, and 49 are already marked complete, representing roughly 61% of the total.
The phase mix for BioMarin Pharmaceutical reports 25 late-stage studies (Phase 3 and Phase 4 combined) and 36 earlier-phase studies (Phase 1 and Phase 2).
The top therapeutic focus area indexed for BioMarin Pharmaceutical is Phenylketonuria (PKU) with 6 linked trials, and 9 other condition areas appear in the top list above.
How to read these pipeline numbers
Recruiting share is one of the more practical signals here: it reflects how much of a sponsor's research is presently open to new participants, while the completed share indicates the depth of finished work that has already contributed registry results. Both counts come directly from the public ClinicalTrials.gov dataset and are refreshed on the registry side; this page mirrors the latest data pull without altering it.
A portfolio weighted toward Phase 3 usually reflects an organization advancing candidates toward regulatory review, where the research centers on comparative efficacy and broader safety across larger populations. A heavier Phase 1 and Phase 2 tilt generally indicates exploratory work, safety, dosing, and early signal detection, and is common among research-forward sponsors that seed many early programs. Phase 4 entries, when present, track interventions already in real-world use and typically focus on long-term safety, effectiveness across subgroups, or formulation comparisons.
That distribution is a quick read of where the organization concentrates its research attention; it does not imply product availability, market share, or any clinical endorsement. All numbers on this page come from ClinicalTrials.gov maintained by the National Library of Medicine, and counts can shift as new studies are registered or existing ones update their status. This information is provided for reference and educational purposes only, not as medical, investment, or regulatory advice, verify current details directly with ClinicalTrials.gov before relying on any figure here.
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial from this sponsor, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.