Feb 27 (Reuters) – The U.S. Food and Drug Administration on Friday approved Ascendis Pharma’s 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.
(Reporting by Sahil Pandey Puyaan Singh and Sneha S K in Bengaluru; Editing by Tasim Zahid)
Comments