BioMarin drug becomes first US approved treatment for dwarfism

Signage can be seen outside the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, USA on August 29, 2020. REUTERS / Andrew Kelly

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Nov. 19 (Reuters) – Daily injection of BioMarin Pharmaceutical Inc (BMRN.O) for children with the most common type of dwarfism received clearance from the US health regulator on Friday, making it the first treatment approved for achondroplasia in the country.

The approval of Voxzogo, aimed at children aged five and over, comes months after it became the first treatment to receive the green light from regulators for the treatment of the genetic disease in Europe.

Many countries still do not have an approved treatment for achondroplasia, a condition characterized by short stature that occurs in about one in 25,000 newborns worldwide.

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BioMarin expects annual net therapy income per patient in the United States to be approximately $ 240,000 and expects approximately 3,000 children with achondroplasia nationwide to be eligible for treatment . Voxzogo is expected to be available in the United States by mid-December at the list price of $ 899 per vial, the company said.

BioMarin’s late stage Voxzogo study showed improvements in patient growth rate over one year and was well tolerated. The drug is also being studied in children under five and has already been approved in Europe for those aged two and over.

In April, the U.S. Food and Drug Administration extended the drug’s review by three months to assess two-year data on the drug.

It has now cleared the drug through the fast-track approval path, which requires a post-market study evaluating final adult height.

BioMarin said it intends to use the results of ongoing extension studies to receive final approval.

The drug is also under regulatory review in Japan, Brazil and Australia and could be approved in those countries next year, BioMarin said.

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Reporting by Amruta Khandekar; Editing by Aditya Soni and Vinay Dwivedi

Our Standards: Thomson Reuters Trust Principles.


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