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 1 of 2

RECRUITING Phase 4

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

NCT07477691

RECRUITING Phase 2

Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia

NCT07441876

RECRUITING Phase 2

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

NCT07126262

RECRUITING Phase 1

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

NCT06738017

RECRUITING Phase 4

Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq

NCT06780332

RECRUITING Phase 2

A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

NCT06668805

RECRUITING Phase 2

A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

NCT06382155

RECRUITING

A Study to Assess Growth in Children With Idiopathic Short Stature

NCT06309979

RECRUITING

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

NCT06305234

RECRUITING

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

NCT06212947

RECRUITING

VIrtual STudy in Achondroplasia for the US (VISTA)

NCT06168201

RECRUITING

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

NCT05579548

RECRUITING

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

NCT05813678

ACTIVE NOT RECRUITING Phase 3

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

NCT06455059

ACTIVE NOT RECRUITING Phase 3

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837

ACTIVE NOT RECRUITING Phase 1

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

NCT05121376

ACTIVE NOT RECRUITING Phase 1

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors

NCT04684940

ACTIVE NOT RECRUITING Phase 1

AAV Gene Therapy Study for Subjects with PKU

NCT04480567

ACTIVE NOT RECRUITING

Cerliponase Alfa Observational Study in the US

NCT04476862

ACTIVE NOT RECRUITING Phase 2

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

NCT03989947

ACTIVE NOT RECRUITING Phase 3

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

NCT03424018

ACTIVE NOT RECRUITING Phase 2

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

NCT02724228

TERMINATED Phase 1

Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants

NCT05813314

COMPLETED Phase 3

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098

COMPLETED

A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A

NCT04560933

COMPLETED

A Prospective Clinical Study of Phenylketonuria (PKU)

NCT04452513

COMPLETED Phase 3

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

NCT03694353

COMPLETED Phase 2

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

NCT03583697

COMPLETED

A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

NCT03505125

COMPLETED Phase 3

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

NCT03392974

COMPLETED Phase 3

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

NCT03370913

COMPLETED Phase 3

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

NCT03197766

COMPLETED Phase 2

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689

COMPLETED

A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302

NCT02468570

COMPLETED Phase 1

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

NCT02485899

TERMINATED

A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)

NCT02191917

COMPLETED

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

NCT02294877

TERMINATED Phase 3

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

NCT01924845

COMPLETED Phase 2

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

NCT02055157

COMPLETED Phase 1

A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease

NCT01907087

COMPLETED Phase 3

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

NCT01889862

TERMINATED Phase 3

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

NCT01803412

COMPLETED Phase 3

An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

NCT01819727

TERMINATED

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

NCT01753804

TERMINATED Phase 2

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

NCT01697319

COMPLETED Phase 2

A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks

NCT01560286

COMPLETED

A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia

NCT01603095

TERMINATED Phase 2

Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome

NCT01609062

COMPLETED Phase 1

A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers

NCT01590446

COMPLETED Phase 2

Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01515956

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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.