See Enlarged Marketed And Pipeline Panel
Our Marketed Programs Include:
PALYNZIQ® (pegvaliase-pqpz) Injection self-administered enzyme substitution therapy for PKU
Additional information is available at: https://www.palynziq.com/
BRINEURA® (cerliponase alfa) for CLN2 Disease
Additional information is available at: https://www.brineura.com/
VIMIZIM® (elosulfase alfa) for MPS IVA
Additional information is available at: https://www.vimizim.com/
KUVAN® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution for PKU
Additional information is available at: https://www.kuvan.com/
NAGLAZYME® (galsulfase) for MPS VI
Additional information is available at: https://www.naglazyme.com/
Our Investigational Programs Include:
Vosoritide (BMN 111): Analog of CNP for Achondroplasia
Valoctocogene Roxaparvovec (BMN 270): AAV-Gene Therapy for Hemophilia A
BMN 307: AAV-Gene Therapy for Phenylketonuria (PKU)
BMN 331: AAV-Gene Therapy for Hereditary Angioedema (HAE)
DiNA-001: AAV-Gene Therapy for Hypertrophic Cardiomyopathy (HCM)
If you have any questions regarding our marketed or investigational programs, please contact BioMarin Global Medical Information by submitting a request via our web request form located here: Web Request Form