MK

Marni Kottle

Executive Vice President, Chief Corporate Affairs Officer

BioMarin Pharmaceutical

BioMarin Pharmaceutical Pipeline

DrugIndicationPhase
VOXZOGO (vosoritide)AchondroplasiaCommercial
PALYNZIQ (pegvaliase-pqpz)Phenylketonuria (PKU)Commercial
Enzyme Replacement TherapyMucopolysaccharidosis ICommercial
Gene TherapyHemophilia ACommercial
Metabolic TherapyRare Metabolic ConditionCommercial
ENPP1 deficiency programENPP1 deficiency/GACIClinical