BioMarin Pharmaceutical Inc. is a biotechnology company that focuses on developing innovative therapies for rare genetic conditions. The company has successfully brought eight marketed products to the market, targeting life-threatening diseases such as achondroplasia, severe hemophilia A, and various lysosomal storage disorders. BioMarin employs a range of treatment modalities, leveraging its scientific expertise to translate genetic discoveries into transformative medicines. The company is also advancing a robust pipeline of investigational medicines, which are currently in various stages of clinical and preclinical development, reflecting its commitment to addressing unmet medical needs in the field of genetic medicine.

The company emphasizes a thoughtful approach to clinical trial design, prioritizing participant safety while efficiently evaluating the safety and efficacy of its investigational medicines. BioMarin conducts clinical trials across multiple phases, from initial safety assessments in healthy volunteers to larger studies that confirm effectiveness and safety in patients with specific conditions. The company is dedicated to ensuring diversity in its clinical trials, aiming to include a broad range of individuals to enhance the applicability of its therapies. This commitment to rigorous research and development underpins BioMarin's mission to improve patient outcomes through innovative genetic therapies.

United States

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.

Cardiovascular Diseases

Central Nervous System (CNS)

Genetic Diseases

Hematologic Diseases

Metabolic Diseases

Musculoskeletal Diseases

Biomarin Pharmaceutical Inc.

Hemophilia

ROCTAVIAN (Valoctocogene roxaparvovec)

Achondroplasia

Genetic Short Stature Conditions

VOXZOGO (vosoritide)

Phenylketonuria

PALYNZIQ® (pegvaliase-pqpz)

Hypochondroplasia

VOXZOGO

Novato, California, United States of America

California - North

California

Adeno-associated viral vector (AAV)

Biologics

Cell Therapies

Gene Therapy

Lentiviral Vector (LVV)

Oligonucleotides

Small Molecule Pharmaceuticals

Therapeutic

Biomarin Pharmaceutical Inc.

Company Details