Europe Approves Biomarin Drug for Type of Dwarfism in Regions First

(Reuters) – U.S.-based Biomarin Pharmaceutical Inc said on Friday the European Commission cleared its treatment for one of the most common forms of dwarfism, the first such approval in the region for a therapy to treat achondroplasia.

The approval for children above two years comes months after the European Medicines Agency endorsed the drug. The U.S. regulators are due to decide on the drug by Nov. 20.

Achondroplasia occurs in about one in 25,000 newborns and is the most common form of disproportionate short stature in humans. It is caused by a mutation in a gene that regulates bone growth.

In clinical trials, the drug was shown to help boost height in children and generally well-tolerated.

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