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

Vosoritide is being tested in children whose growth plates are still “open,” typically those under 18 years of age. This is approximately 25 percent of people with achondroplasia. In the US, Europe, Latin America, the Middle East and APAC (except for Japan), there are currently no licensed medications for achondroplasia.

Achondroplasia is characterized by failure of normal conversion of cartilage into bone, which results in impaired bone growth. This condition is caused by a change in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth. Beyond disproportionate short stature, people with achondroplasia can experience serious health complications, including foramen magnum compression, sleep apnea, bowed legs, mid-face hypoplasia, permanent sway of the lower back, spinal stenosis and recurrent ear infections. Some of these complications can result in invasive surgeries such as spinal cord decompression and straightening of bowed legs. In addition, some studies show increased mortality at every age.

More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births.


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.


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