BioMarin Pharmaceutical
Open enrollment across a mixed-phase pipeline
According to ClinicalTrials.gov-derived indexes, BioMarin Pharmaceutical sits in the top decile of US sponsors by registered trial count (#280 of 15,742 sponsors by registered trial count).
BioMarin Pharmaceutical: 80 sponsored US clinical trials, 13 recruiting.
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.
Top-decile sponsor footprint
BioMarin Pharmaceutical ranks in the top decile of sponsors by registered trial count (#280 of 15,742 sponsors by registered trial count). Volume at this scale usually reflects a long institutional pipeline, read phase tilt and therapeutic areas below before treating it as current-year activity.
Trial completion reliability score
49 of 58 decided trials (Completed vs. Terminated on ClinicalTrials.gov) reached their planned completion (84.5%), vs. national p10 68.4% / p90 100% among 1,234 sponsors with at least 10 decided trials. Still-open trials (Recruiting, Active-not-recruiting, Not-yet-recruiting) are excluded since they have not reached an outcome yet.
Open and recent trials
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 is open in the BioMarin Pharmaceutical pipeline
13 recruiting of 80 indexed (16% open); 49 completed (61%).
Phase mix: 25 late (III/IV) vs 36 early (I/II) (balanced across stages).
Top condition: Phenylketonuria (PKU) (6 trials), 9 others listed.
How open-pipeline share is computed
Recruiting share is recruiting_count divided by trial_count for this sponsor in the public registry export. Status can change between pulls; treat the figure as a research-attention snapshot, not product availability.
BioMarin Pharmaceutical: open-pipeline peers nationwide
Peers matched on 80 registered trials and 16% open enrollment, not the therapeutic-area sidebar.
BioMarin Pharmaceutical sits below the median trial volume and at or above the median recruiting share among these 9 nationwide peers -- the two do not always move together.
Sponsors near 80 trials (± recruiting share)
- Children's Healthcare of Atlanta · 80 trials · 3% recruiting
- EMD Serono Research & Development Institute · 81 trials · 11% recruiting
- The University of Tennessee, Knoxville · 81 trials · 7% recruiting
- TriHealth · 81 trials · 2% recruiting
Sponsors near 16% open enrollment (± trial volume)
- Atlantic Health System · 16% recruiting · 43 trials
- National Human Genome Research Institute (NHGRI) · 16% recruiting · 185 trials
- Drexel University · 16% recruiting · 92 trials
- Case Western Reserve University · 16% recruiting · 141 trials
Related
What to do with this BioMarin Pharmaceutical page
80 registered trials is a research-attention snapshot, not an endorsement of BioMarin Pharmaceutical or any single study.
- 13 of BioMarin Pharmaceutical's trials are currently recruiting, check each one's own eligibility criteria before contacting a site. Browse recruiting trials
- Phenylketonuria (PKU) is the condition BioMarin Pharmaceutical studies most, compare against other sponsors active in the same condition before evaluating a specific study. See Phenylketonuria (PKU) trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
BioMarin Pharmaceutical pipeline card: 80 registered trials from official public datasets.Report a data error.