UPDATE 1-Ascendis wins U.S. approval for therapy for children with dwarfism

BioMarin Pharmaceutical Inc. -3.04%
BridgeBio Pharma -1.75%
Bank of New York Mellon Corp +0.96%

BioMarin Pharmaceutical Inc.

BMRN

55.50

-3.04%

BridgeBio Pharma

BBIO

73.15

-1.75%

Bank of New York Mellon Corp

BK

122.13

+0.96%

Adds details throughout

- 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.