MK

Marni Kottle

Executive Vice President, Chief Corporate Affairs & Patient Advocacy 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