© 2020 BioMarin. All rights reserved.

More than 20 years developing therapies for rare genetic disorders.

Each therapy we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it is designed to address. The result: six first or best-in-class treatments with another four in development.

Molecule
Condition
Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)
Image: Lab technician filling vial
Our Products

We find tomorrows.

Each of our approved therapies were driven by the goal of resetting the standard of care for the conditions they treat.

Learn More about our products
Our Pipeline

We take on the biggest challenges in rare disease.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.

View Now Our Pipeline
Boy on swing
Compassionate Use Policy

We are driven by patient need.

We are committed to developing safe and effective therapies and providing those therapies to the patients as quickly as possible.

Compassionate Use Policy about our Compassionate Use Policy