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BioMarin drug becomes the first drug approved in the United States for the treatment of dwarfism

By: jack an Nov. 22,2021
BioMarin Pharmaceutical Inc (BMRN.O)'s once-daily injection for the treatment of children with the most common form of dwarfism was approved by U.S. health regulators on Friday, making it the first drug approved for the treatment of chondrodysplasia in the country.

Voxzogo's approval comes just months after it became the first therapy to be approved by regulators for the treatment of a genetic disorder in Europe for children 5 years and older.

Many countries still do not have approved therapies for chondrodysplasia, a disorder characterized by short stature that occurs in about one in 25,000 births worldwide.

BioMarin expects the therapy to net about $240,000 per patient per year in the U.S. and expects about 3,000 children with chondrodysplasia to be eligible for the treatment in that country. Voxzogo is expected to be available in the U.S. in mid-to-late December, priced at $899 per vial, the company said.

BioMarin's late-stage Voxzogo study showed that patients' growth rates improved within a year and were well tolerated. The drug is also being studied in children under 5 years of age and has been approved in Europe for use in children 2 years and older.

The FDA extended its review of the drug by three months in April to evaluate two years of data on the drug.

It has now cleared the drug through the accelerated approval pathway, which requires a post-marketing study evaluating final adult height.

BioMarin said it intends to use the results of the ongoing extension study to gain final approval.

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