By Colin Kellaher


Dyne Therapeutics Inc. on Thursday said the U.S. Food and Drug Administration granted a pair of key designations to its DYNE-251 investigational therapeutic for certain patients with Duchenne muscular dystrophy, or DMD.

The Waltham, Mass., clinical-stage muscle-disease company said the FDA granted orphan-drug and rare-pediatric-disease designations to DYNE-251, which it is evaluating in a Phase 1/2 study in patients with DMD with mutations amenable to skipping exon 51.

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.

DMD, a rare, fatal neuromuscular genetic disease that occurs in roughly one in every 3,500 to 5,000 males, is caused by a change or mutation in the gene that encodes instructions for dystrophin, a protein found in muscle cells. There is no cure for DMD, and currently approved therapies provide limited benefit.

Dyne said it expects to report initial data from the multiple ascending dose placebo-controlled portion of the Phase 1/2 study of DYNE-251 in the second half of the year.


Write to Colin Kellaher at


(END) Dow Jones Newswires

March 23, 2023 08:04 ET (12:04 GMT)

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