Biomarin Pharmaceutical Inc.
- Address
- 105 Digital Drive
Sponsor Overview
Specializations
Growth Disorders and Skeletal Dysplasias
BioMarin Pharmaceutical Inc. focuses heavily on conditions affecting linear growth and bone development, including achondroplasia, hypochondroplasia, idiopathic short stature, Turner syndrome, short stature homeobox-containing gene deficiency, and Noonan syndrome. Its research also extends to pediatric growth impairment linked to inadequate response to human growth hormone.
- Achondroplasia
- Hypochondroplasia
- Idiopathic Short Stature
- Turner Syndrome
- Noonan Syndrome
The sponsor’s clinical activity in this area includes children, infants, and adolescents, with a strong emphasis on growth-related outcomes and long-term treatment experience.
Duchenne Muscular Dystrophy
BioMarin also invests in research for Duchenne muscular dystrophy, a progressive neuromuscular disorder associated with severe functional decline. The clinical portfolio reflects interest in therapies aimed at improving disease management in affected children and young people.
- Duchenne Muscular Dystrophy
- Neuromuscular disease
- Pediatric treatment
This area highlights the company’s involvement in therapeutic development for inherited disorders with substantial unmet medical need.
Hemophilia A and Bleeding Disorders
Another key therapeutic area is hemophilia A, where BioMarin supports clinical research related to long-term management and follow-up in patients with inherited bleeding disorders. The focus is on maintaining safety and treatment experience in individuals previously exposed to gene-based or factor-directed approaches.
- Hemophilia A
- Inherited bleeding disorders
- Long-term follow-up
These studies place the sponsor within the field of rare hematologic disease research.
Phenylketonuria and Metabolic Disorders
BioMarin’s research portfolio includes phenylketonuria, a rare metabolic condition requiring ongoing dietary and pharmacologic management. The company’s work in this area reflects interest in improving treatment options for adolescents living with inherited amino acid metabolism disorders.
- Phenylketonuria
- Metabolic disease
- Adolescent care
This therapeutic domain broadens the sponsor’s rare disease focus beyond growth and neuromuscular conditions.
Sponsored Trials
Data & Insights
This sponsor works with 3 molecules, of which 3 are not being studied by other sponsors.
This sponsor has worked with 41 sites across their trial portfolio.
Therapeutic Focus
- 1.Congenital, Hereditary, and Neonatal Diseases and Abnormalities3 trials165 other sponsors
- 2.Musculoskeletal Diseases3 trials238 other sponsors
- 3.Musculoskeletal and Neural Physiological Phenomena2 trials28 other sponsors
- 4.Genetic Phenomena1 trial26 other sponsors
- 5.Hemic and Lymphatic Diseases1 trial249 other sponsors
- 6.Hormonal diseases1 trial89 other sponsors
- 7.Nutritional and Metabolic Diseases1 trial211 other sponsors
Partnered Research Centres
41 centres- Address
- 264 Boulevard de Saint Pierre, 13005 Marseille
- Address
- 264 Rue Saint Pierre, 13005 Marseille
- Address
- Viale Gaetano Pieraccini 24, 50139 Florence
- Address
- Piazza Luigi Miraglia 2, 80138 Naples
- Address
- 78 Rue Du General Leclerc, 94275 Le Kremlin-Bicetre
- Address
- Langenbeckstrasse 1, Oberstadt, 55131 Mainz
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