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PepGen Receives U.S. FDA Orphan Drug and Rare Pediatric Disease Designations for PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy
BOSTON, March 13, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide
About this update from Pepgen Inc.
[{"type":"text","content":"BOSTON, March 13, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) granted both orphan drug and rare pediatric disease designations for PGN-EDO51, an investigational therapeutic for Duchenne muscular dystrophy (DMD) patients whose mutations are amenable to an exon 51 skipping approach. PepGen is evaluating PGN-EDO51 for the treatment of DMD in the ongoing CONNECT1 Phase 2 trial, and expects to begin enrolling patients in the CONNECT2 Phase 2 trial later this year. “Receiving these FDA designations for PGN-EDO51 emphasizes the critical demand for novel and enhanced therapeutic options for people living with DMD,” said James McArthur, Ph.D., President and CEO of PepGen. “With our CONNECT1 Phase 2 trial underway, we look forward to reporting preliminary data this year.” Orphan designation is granted by the FDA to advance the evaluation and development of new treatments intended to treat a rare disease or condition, which is generally a disease or condition that affects either (i) fewer than 200,000 individuals in the United States, or (ii) more than 200,000 individuals on the basis of certain facts and circumstances. Under the Orphan Drug Act, the FDA may provide sponsors incentives including tax credits for qualified clinical trials, FDA user-fee benefits, and seven years of market exclusivity in the United States after approval. The FDA defines a \"rare pediatric disease\" as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under the Rare Pediatric Disease program, the FDA may award priority review vouchers to sponsors who have been granted rare pediatic disease designation for its drug. A sponsor who has been granted the designation and receives an approval for a drug for a rare pediatric disease may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. For more information on these designations, please visit the FDA website’s Orphan Drug and Rare Pediatric Disease webpages. About PGN-EDO51 ...