PRELOAD PRELOAD PRELOAD PRELOAD PRELOAD

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 bar to learn more about each product or product candidate.

Aldruazyme for MPS I Naglazyme for MPS VI Kuvan for PKU Firdapse for LEMS VIMIZIM for Morquio A Syndrome PEG-PAL for PKU BMN-673: PARP Inhibitor for Genetically Defined Cancers BMN-701-IGF-GAA-for-Pompe-Disease BMN-111-CNP-for-Achondroplasia BMN-190-TPP1-for-CLN2 BMN-270-AAV-Factor VIII Vector for Hemophilia A BMN-250-GILT rhNAGLU for Sanfilippo Syndrome



*MBC=metastatic breast cancer
*SCLC=small cell lung cancer