Solid Biosciences Gets FDA Rare-Pediatric Designation for SGT-003 in Duchenne

By Colin Kellaher

Solid Biosciences has won U.S. Food and Drug Administration rare-pediatric-disease designation for its SGT-003 gene-therapy candidate for Duchenne muscular dystrophy.

The Charlestown, Mass., life-science company on Monday said site activation is scheduled this month for a Phase 1/2 first-in-human, open-label study to determine the safety and tolerability of SGT-003 in pediatric patients with Duchenne, a rare, fatal neuromuscular genetic disease that occurs in roughly one in every 3,500 to 5,000 males.

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.

Solid said it expects to begin dosing patients in the study in the second quarter.

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

(END) Dow Jones Newswires

April 01, 2024 08:14 ET (12:14 GMT)

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