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Brian R. Mueller

Executive Vice President, Chief Financial Officer

BioMarin Pharmaceutical

BioMarin Pharmaceutical Pipeline

DrugIndicationPhase
VOXZOGO (vosoritide)AchondroplasiaApproved/Marketed
ROCTAVIAN (valoctocogene roxaparvovec)Severe Hemophilia AApproved/Marketed
PALYNZIQ (pegvaliase-pqpz)Phenylketonuria (PKU)Approved/Marketed
BMN 331 (giroctocogene fitelparvovec)Severe Hemophilia BPhase 1/2
BMN 349Genetic CardiomyopathyPreclinical
BMN 355Undisclosed Genetic CNS DisorderPreclinical