PRELOAD PRELOAD PRELOAD PRELOAD PRELOAD

    Clinical Development

    By streamlining clinical and regulatory development, BioMarin is providing rapid access to treatment to patients around the world suffering from rare inherited genetic diseases. The company is focused on accelerating the regulatory approval process by maximizing integrated product development strategies and leveraging the expertise of collaborative, cross-functional teams of clinical and regulatory professionals. Currently, BioMarin has an exciting range of breakthrough drug candidates now in clinical development. A variety of other promising preclinical programs are also being pursued with the goal of filing one IND per year to ensure continued growth.

    Click on indications to learn more about each product candidate.

    GALNS-for-MPS-IVA-Morquio-A-Syndrome PEG-PAL for PKU BMN-701-IGF-GAA-for-Pompe-Disease BMN-673-PARP-Inhibitor-for-Genetically-Defined-Cancers