UPDATE 1-Ascendis wins U.S. approval for therapy for children with dwarfism
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Feb 27 (Reuters) - The U.S. Food and Drug Administration on Friday approved Ascendis Pharma's A71.F once-weekly therapy for children with a rare genetic disorder that causes dwarfism, the company said.
The treatment, branded Yuviwel, is a once-weekly injection designed to provide sustained exposure to C-type natriuretic peptide, which Ascendis said can counter growth-limiting effects driven by the FGFR3 mutation in achondroplasia.
Ascendis said continued approval for Yuviwel may depend on confirmation of clinical benefit in post-approval trials.
Achondroplasia is the most common type of short-limbed dwarfism. The condition occurs in 1 in 15,000 to 40,000 newborns, according to the National Institutes of Health.
The disorder is caused by a genetic mutation that affects a protein in the body called fibroblast growth factor receptor 3, or FGFR3, resulting in dwarfism.
Ascendis said it plans to launch Yuviwel in the United States in early Q2 2026.
