Biomarin Pharmaceutical
- Country
- 🇨🇦Canada
- Ownership
- -
- Established
- 1997-01-01
- Employees
- -
- Market Cap
- $17.2B
- Website
- http://www.biomarin.com
Clinical Trials
108
Trial Phases
5 Phases
Drug Approvals
0
Drug Approvals
No drug approvals found
This company may not have drug approvals in our database
Clinical Trials
Distribution across different clinical trial phases (78 trials with phase data)• Click on a phase to view related trials
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
- First Posted Date
- 2025-08-17
- Last Posted Date
- 2025-08-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 60
- Registration Number
- NCT07126262
- Locations
- 🇺🇸
Phoenix Children's Hospital - Thomas Campus (Main), Phoenix, Arizona, United States
🇺🇸Cedars-Sinai Medical Center, Los Angeles, California, United States
🇺🇸Benioff Children's Hospital - Oakland, Oakland, California, United States
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
- First Posted Date
- 2025-07-18
- Last Posted Date
- 2025-07-18
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 140
- Registration Number
- NCT07073014
- Locations
- 🇺🇸
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children), Wilmington, Delaware, United States
🇺🇸Children's National Medical Center, Washington, District of Columbia, United States
🇺🇸Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq
- Conditions
- PhenylketonuriaPKU
- Interventions
- Drug: RDD to Palynziq
- First Posted Date
- 2025-01-17
- Last Posted Date
- 2025-01-23
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 10
- Registration Number
- NCT06780332
- Locations
- 🇺🇸
Uncommon Cures, Chevy Chase, Maryland, United States
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
- First Posted Date
- 2024-12-17
- Last Posted Date
- 2025-07-24
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 12
- Registration Number
- NCT06738017
- Locations
- 🇬🇧
University Hospital Southampton NHS Foundation Trust, Southampton, UK, United Kingdom
🇺🇸University of California, San Diego, San Diego, California, United States
🇺🇸Saint Louis University, St. Louis, Missouri, United States
A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone
- Conditions
- Short Stature Homeobox- Containing Gene SHOX DeficiencyNoonan SyndromeTurner Syndrome
- Interventions
- First Posted Date
- 2024-10-31
- Last Posted Date
- 2025-08-06
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 72
- Registration Number
- NCT06668805
- Locations
- 🇺🇸
Children's National Medical Center, Washington DC, District of Columbia, United States
🇫🇷South Paris University Hospitals - Bicetre Hospital, Le Kremlin-Bicêtre, Paris, France
🇫🇷Hôpital Robert-Debré, Paris Cedex 19, Paris, France
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News
Aurobindo Pharma Secures Multiple FDA Approvals for Generic Drug Portfolio Expansion
Aurobindo Pharma Limited has received FDA approval for multiple generic drug formulations, including critical medications for cardiovascular, neurological, and gastrointestinal conditions.
Spruce Biosciences Acquires Tralesinidase Alfa for Sanfilippo Syndrome Type B Treatment with BLA Submission Planned for 2026
Spruce Biosciences has acquired tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B, a neurodegenerative and fatal genetic disease with no FDA-approved treatments.
Phenylketonuria Pipeline Shows Robust Growth with 25+ Therapies in Development
DelveInsight's latest report reveals a robust phenylketonuria (PKU) pipeline with over 20 companies developing 25+ therapeutic candidates, signaling significant advancement in treatment options for this rare metabolic disorder.
Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments
Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.
AAV Gene Therapy Market Projected to Reach $107.2 Billion by 2035, Growing at 40% CAGR
• The adeno-associated virus (AAV) gene therapy market is expected to surge from $2.7 billion in 2024 to $107.2 billion by 2035, driven by advances in genetics, biotechnology, and personalized medicine. • Marketed AAV therapies include Luxturna for hereditary retinal dystrophy, Zolgensma for spinal muscular atrophy, and Glybera for lipoprotein lipase deficiency, with numerous promising candidates in late-stage development. • Improved diagnostic technologies, regulatory support, and significant investment from companies like Spark Therapeutics, Novartis, and BioMarin are accelerating the development of next-generation AAV therapies for previously untreatable genetic disorders.