Edgewise Gets FDA Orphan, Rare-Pediatric-Disease Designations for Muscular-Dystrophy Program

By Colin Kellaher

Edgewise Therapeutics has won a pair of key U.S. Food & Drug Administration designations for its EDG-5506 muscular-dystrophy program.

The Boulder, Colo., clinical-stage biopharmaceutical company on Thursday said the FDA granted orphan-drug designation to EDG-5506 for the treatment of Duchenne and Becker muscular dystrophy, and rare-pediatric-disease designation for the treatment of Duchenne.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

The agency awards priority-review vouchers to companies upon approval of drugs that are granted the rare-pediatric-disease designation, and those vouchers can be used to obtain priority review for another drug or sold to other companies.

Edgewise is currently advancing EDG-5506, a small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Duchenne and Becker, in multiple Phase 2 studies.

There are currently no cures for Duchenne or Becker muscular dystrophy, which are neuromuscular genetic diseases.

Write to Colin Kellaher at [e-mail]

(END) Dow Jones Newswires

November 30, 2023 08:41 ET (13:41 GMT)

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