Company intelligence / Profile preview

BioMarin Pharmaceutical

Ownership: Private
Employees: ~3000
Development stage: Other

Overview

BioMarin has eight approved drugs, including enzyme replacement therapies and the first gene therapy for hemophilia A, with robust clinical trials ongoing and recent strong pivotal results for PALYNZIQ in adolescent PKU. The company is a global leader in rare genetic diseases, with a revenue of around $2.7 billion in 2024, and a strategy of rapidly advancing high-impact programs through clinical development.[1][3][6][8]

Therapeutic areas: HematologyNeurologyMetabolic DisordersRare Diseases
Modalities: Gene therapyEnzyme replacement therapySmall moleculeRecombinant proteinBiologic
Industry: Biotech

Drug pipeline

11 assets

sapropterin

Approved

10 more assets in the full pipeline.

Partnerships

Genzyme (Aldurazyme co-development)University College London/St. Jude Children’s Research Hospital (Roctavian gene therapy licensing)

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