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 1 of 2
Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
NCT07477691
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
NCT07441876
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
NCT07126262
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
NCT06738017
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
NCT06780332
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
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
NCT06382155
A Study to Assess Growth in Children With Idiopathic Short Stature
NCT06309979
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
NCT06305234
A Multicenter Multinational Observational Study of Children With Hypochondroplasia
NCT06212947
VIrtual STudy in Achondroplasia for the US (VISTA)
NCT06168201
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
NCT05579548
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
NCT05813678
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
NCT06455059
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT05270837
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
NCT05121376
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
NCT04684940
AAV Gene Therapy Study for Subjects with PKU
NCT04480567
Cerliponase Alfa Observational Study in the US
NCT04476862
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT03989947
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03424018
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT02724228
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
NCT05813314
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
NCT04323098
A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
NCT04560933
A Prospective Clinical Study of Phenylketonuria (PKU)
NCT04452513
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
NCT03694353
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
NCT03583697
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT03505125
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
NCT03392974
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
NCT03370913
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
NCT03197766
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT02678689
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
NCT02468570
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
NCT02485899
A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
NCT02191917
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
NCT02294877
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
NCT01924845
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT02055157
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
Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT01889862
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT01803412
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
NCT01819727
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
NCT01753804
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
NCT01697319
A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks
NCT01560286
A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia
NCT01603095
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
NCT01609062
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
NCT01590446
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
NCT01515956
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.