BioMarin Pharmaceutical Inc.  

Novato,  CA 
United States
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Welcome to the BioMarin Global Medical Information Booth.


BioMarin is a world leader in developing and commercializing innovative therapies for rare diseases driven by genetic causes. With a 20-year history, BioMarin remains steadfast to its original mission - to bring new treatments to market that will make a big impact on small patient populations. These conditions are often inherited, difficult to diagnose, progressively debilitating, have few, if any, treatment options, and are usually ignored.  Visit to learn more.


  • Global Medical Information Virtual Booth
    For full prescribing information, including boxed warnings for PALYNZIQ® (pegvaliase-pqpz) and VIMIZIM® (elosulfase alfa), please see full prescribing information available at the product websites listed below....

  • 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:
    BRINEURA® (cerliponase alfa) for CLN2 Disease

    Additional information is available at:
    VIMIZIM® (elosulfase alfa) for MPS IVA

    Additional information is available at:
    KUVAN® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution for PKU

    Additional information is available at:
    NAGLAZYME® (galsulfase) for MPS VI

    Additional information is available at:

    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