Salem Radio Network News Wednesday, December 8, 2021


BioMarin wins first U.S. drug approval for common type of dwarfism

(Reuters) -BioMarin Pharmaceutical Inc’s injection for children with the most common type of dwarfism received clearance from the U.S. health regulator on Friday, making it the first approved therapy for achondroplasia in the country.

The approval for Voxzogo, intended to improve growth in children aged five and older, comes months after it became the first approved therapy for the disorder 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 drugmaker’s shares jumped more than 6%, on course to erase all the losses suffered this year.

The approval was based on results from a late-stage study that showed a statistically significant improvement in the growth rate of children with achondroplasia treated with Voxzogo over one year.

The U.S. Food and Drug Administration has cleared the drug under the accelerated approval pathway, which requires a post-marketing study assessing final adult height.

BioMarin said it intended to use results from ongoing extension studies for the post-marketing requirement.

The company expects to make Voxzogo available in the United States by mid-to-late December.

The drug is also under regulatory review in Japan, Brazil, and Australia and could receive approval in the countries next year, BioMarin said.

(Reporting by Amruta Khandekar; Editing by Aditya Soni)


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