The FDA approved vosoritide (Voxzogo) to treat kids ages 5 years and older with achondroplasia, the most common form of dwarfism.
Achondroplasia is caused by a genetic mutation that leads to overactivity of fibroblast growth factor receptor 3, which prevents normal bone growth and leads to “severely short stature and disproportionate growth,” the agency explained. Vosoritide—a once-daily subcutaneous injection that comes in 0.4 mg, 0.56 mg, or 1.2 mg doses based on the patient’s weight—works by binding to natriuretic peptide receptor-B to reduce the gene’s activity and stimulate bone growth. However, the agency noted that vosoritide is only indicated for children with open epiphyses, meaning they still have the potential to grow.
“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, MD, director of the Division of General Endocrinology in the FDA’s Center for Drug Evaluation and Research, in a statement. “With this action, children with short stature due to achondroplasia have a treatment option that targets the underlying cause of their short stature.”
This approval was based on results from a one-year, double-blind, placebo-controlled phase III study in patients 5 years and older with achondroplasia with open epiphyses. “In the study, 121 participants were randomly assigned to receive either [vosoritide] injections under the skin or a placebo. Researchers measured the participants’ annualized growth velocity, or rate of height growth, at the end of the year. Participants who received [vosoritide] grew an average 1.57 centimeters taller compared to those who received a placebo.”
The most common adverse events related to use of vosoritide were injection site reactions, vomiting, and decreased blood pressure, the agency warned.
Vosoritide is manufactured by BioMarin.
John McKenna, Associate Editor, BreakingMED™
Cat ID: 138
Topic ID: 85,138,730,138,683,192,725,925