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Pipeline

Pipeline

Developing promising new therapies for patients with rare genetic diseases.

BioMarin is actively pursuing strategies to enhance its exciting product portfolio. The company has a growing product pipeline, with multiple clinical and preclinical IND (Investigational New Drug) candidates in development.

Click on the pipeline to learn more about each product candidate.

Naglazyme for MPS VI Aldruazyme for MPS I Kuvan for PKU Firdapse for LEMS 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 BMN-111-CNP-for-Achondroplasia