BioMarin Pharmaceutical
Trial Pipeline
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
A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT03505125
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT02678689
Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
NCT01889862
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 Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
NCT01114737
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 Phase 2 Study of the Effects of Sapropterin Dihydrochloride on Symptomatic Peripheral Arterial Disease
NCT00403494
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
NCT00299000
Study of rhASB in Patients With Mucopolysaccharidosis VI
NCT00104234
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 5 |
| Phase 2 | 8 |
| Phase 3 | 7 |
| Phase 4 | 2 |
What the Pipeline for BioMarin Pharmaceutical Shows
According to the ClinicalTrials.gov registry, BioMarin Pharmaceutical is linked to 31 US clinical trials across every stage of research activity. Of those, 11 studies are currently recruiting — about 35% of the sponsor's indexed portfolio — and 11 are already marked complete, representing roughly 35% of the total. 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.
The phase mix for BioMarin Pharmaceutical reports 9 late-stage studies (Phase 3 and Phase 4 combined) and 13 earlier-phase studies (Phase 1 and Phase 2). 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.
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. 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.