FDA Approves BioMarin's Drug To Treat Dwarfism

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) Friday said that the U.S. Food and Drug Administration has granted accelerated approval to Voxzogo, the first drug approved by the FDA to improve growth in children with most common form of dwarfism.

The drug has been approved to increase linear growth in pediatric patients with achondroplasia five years of age and older with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity.

However, continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory studies. To

Achondroplasia, the most common form of disproportionate short stature in humans, is caused by a mutation in fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth.

"Today's approval fulfills an unmet medical need for more than 10,000 children in the United States and underscores the FDA's commitment to help make new therapies available for rare diseases," said Theresa Kehoe, M.D., director of the Division of General Endocrinology in the FDA's Center for Drug Evaluation and Research. "With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature."

Voxzogo's safety and efficacy in improving growth were evaluated in a year-long phase 3 study in participants five years and older with achondroplasia who have open epiphyses. In the study, 121 participants were randomly assigned to receive either Voxzogo injections under the skin or a placebo. Participants who received Voxzogo grew an average 1.57 centimeters taller compared to those who received a placebo.

Vosoritide was approved under brand name VOXZOGO by the European Commission in August 21, 2021, to treat achondroplasia in children, becoming the first approved drug for this condition in Europe.

According to analysts, Voxzogo could bring in peak sales of over $500 million to $1 billion.

BMRN closed Friday's trading at $91.47, up $8.55 or 10.31%, on the Nasdaq.