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U.S. FDA approves BioMarin’s drug for a type of dwarfism

By Thomson Reuters Nov 19, 2021 | 10:06 AM

(Reuters) – The U.S. Food and Drug Administration (FDA) on Friday approved BioMarin Pharmaceutical Inc’s drug to improve growth in children with a type of dwarfism.

The approval, meant for children five years of age and older, comes months after Voxzogo became the first approved drug for achondroplasia in Europe. Achondroplasia is a genetic disorder affecting bone growth and leads to a short stature.

BioMarin’s drug works by reducing the activity of a gene that prevents normal bone growth in patients with the disorder.

The regulator has cleared the drug under the accelerated approval pathway, which requires the company to conduct a post-marketing study.

(Reporting by Amruta Khandekar; Editing by Aditya Soni)