BioMarin Pharmaceutical

80 total trials 13 currently recruiting 49 completed

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

TERMINATED Phase 2

Extension Study for Patients Who Have Participated in a BMN 701 Study

NCT01435772

COMPLETED Phase 3

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01415427

COMPLETED

Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02

NCT01387854

COMPLETED Phase 2

Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)

NCT01212744

COMPLETED Phase 3

A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01275066

COMPLETED Phase 1

Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease

NCT01230801

COMPLETED Phase 3

Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients

NCT01114737

COMPLETED Phase 2

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

NCT00924703

COMPLETED Phase 2

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

NCT00925054

COMPLETED Phase 3

Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU

NCT00838435

COMPLETED Phase 1

A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects

NCT00789568

TERMINATED

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

NCT00787995

COMPLETED

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

NCT00778206

COMPLETED Phase 1

Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria

NCT00634660

COMPLETED Phase 2

A Phase 2, Pharmacokinetic (PK) Study of 6R-BH4 Alone or 6R-BH4 With Vitamin C in Subjects With Endothelial Dysfunction

NCT00532844

COMPLETED Phase 2

A Phase 2 Study of the Effects of 6R-BH4 in Subjects With Sickle Cell Disease

NCT00445978

COMPLETED Phase 2

A Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease

NCT00403494

COMPLETED Phase 2

Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency

NCT00355264

COMPLETED Phase 3

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

NCT00332189

COMPLETED Phase 4

A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI

NCT00299000

COMPLETED Phase 2

A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension

NCT00325962

COMPLETED Phase 3

Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet

NCT00272792

COMPLETED Phase 3

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

NCT00225615

COMPLETED

Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

NCT00214773

COMPLETED Phase 3

Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

NCT00104247

COMPLETED Phase 2

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

NCT00104260

COMPLETED Phase 3

Study of rhASB in Patients With Mucopolysaccharidosis VI

NCT00104234

COMPLETED Phase 3

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

NCT00067470

COMPLETED Phase 2

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

NCT00048711

COMPLETED Phase 1

Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

NCT00048620

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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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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.