Wave Life Sciences Gets FDA Rare-Pediatric Designation for WVE-N531

By Colin Kellaher

Wave Life Sciences has won Food and Drug Administration rare-pediatric-disease designation for its WVE-N531 drug candidate in the neuromuscular genetic disease Duchenne muscular dystrophy.

The clinical-stage biotechnology company said the designation covers WVE-N531 for the treatment of boys with Duchenne who are amenable to exon 53 skipping.

The FDA grants rare-pediatric-disease designation to drugs aimed at serious or life-threatening diseases that affect fewer than 200,000 people in the U.S. and primarily affect those ages 18 years and younger. The agency awards priority-review vouchers to companies upon approval of certain drugs that are granted the designation, and those vouchers can be used to obtain priority review for another drug or sold to other companies.

Wave said it expects to report data from a potentially registrational clinical trial of WVE-N531 in the third quarter.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

August 12, 2024 09:27 ET (13:27 GMT)

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