Ascendis' drug wins US approval to treat children with dwarfism

The US Food and Drug Administration on Friday approved the drug from Ascendis Pharma. The company said the drug is intended for weekly use in children with a rare genetic disorder that causes dwarfism.

The drug, branded Yuviwel, is an injection called xrust, given once a week, and is designed to provide long-term exposure to C-type natriuretic peptide, which Ascendis says can counteract growth retardation caused by FGFR3 mutation in achondroplasia.

Ascendis said continued approval of Yuviwel may be contingent on confirmation of clinical efficacy in post-marketing studies.

Achondroplasia is the most common type of dwarfism with shortened limbs. The condition occurs in 1 in 15,000 to 40,000 newborns, according to the National Institutes of Health.

This disease is caused by a genetic mutation that affects a protein in the body called fibroblast growth factor receptor 3, or FGFR3, which results in dwarfism.

Ascendis announced plans to launch Yuviwel in the United States early in the second quarter of 2026.

FDA approval was based on results from Ascendis' 52-week study in children ages 2 to 11 years.

Results showed a statistically significant improvement in annual growth rate compared to placebo, the company said. An alternative medicine is the only drug Voxogo.

While Voxzogo requires daily dosing, Ascendis' therapy was designed as a weekly injection therapy.

According to TD Cowen analyst Yaron Verber, approximately 20% of patients do not tolerate Voxzogo well and may consider switching to another drug. Werber estimates peak sales of Juvivel at €772 million in 2032.

Xrust Ascendis' drug received approval in the US for the treatment of children with dwarfism