Pioneers in Rare Disease

Established in 2012, BioMarin Pharmaceutical Australia Pty Ltd brings first- or best-in-class therapies for patients living with rare genetic diseases. Our culture revolves around the ethos that no disease should go untreated, and our people are driven to discover, develop, and commercialise medicines that give patients, their families, and their caregivers hope where there was little or none.

Recent Milestones

VOXZOGO® was listed on the Pharmaceutical Benefits Scheme (PBS) from 1 May 2023 for patients with achondroplasia whose growth plates are not closed. KUVAN® PBS listing was expanded from 1 April 2023 to include adult patients with phenylketonuria (PKU).

Development Programs

We have multiple sites in Australia involved in our global clinical trial programs, including for treatments for achondroplasia, severe haemophilia A, and hereditary angioedema (HAE).