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We seek the big effect.

By strategically focusing on rare disorders with de­fined genetic defects, we strive to develop innovative therapies that are ­first- or best-in-class.

Molecule
Condition
Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)
  • Vosoritide (BMN 111) Analog of CNP
    Achondroplasia
    NDA/MAA
    Target Indication

    Vosoritide is an investigational analog of C-type Natriuretic Peptide under investigation for the treatment of children with achondroplasia, the most common form of skeletal dysplasia.

  • Valoctocogene Roxaparvovec (BMN 270) AAV-Factor VIII
    Severe Hemophilia A
    MAA
    Target Indication

    Valoctocogene roxaparvovec is an investigational AAV5 gene therapy under regulatory review for the treatment of severe hemophilia A.

    Learn More about Valoctocogene Roxaparvovec
  • BMN 307 AAV
    Gene Therapy
    PKU
    Phase 1
    Target Indication

    BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy under investigation to determine if it can normalize blood phenylalanine concentration levels in patients with PKU.

    Learn More
  • BMN 331 AAV
    Gene Therapy
    Hereditary Angiodema
    (HAE)
    Preclinical
    Target Indication

    BMN 331 is a gene therapy candidate in development for Hereditary Angioedema.

    Learn More about BMN 331 AAV
  • BMN 255
    Chronic Renal Disease
    Pre-clinical
    Target Indication

    BMN 255 is an investigational drug candidate in development for chronic renal disease.

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Clinical Trials

More than 20 years of proven clinical success.

Our thoughtful approach to clinical trial design enables us to move fast and efficiently while protecting the safety of our patients.

Learn More about our clinical trials