PepGen's Rare Muscle Disorder Therapy Gets FDA Orphan Drug, Rare Pediatric Disease Designations

By Zaeem Shoaib

PepGen has received the U.S. Food and Drug Administration's orphan drug and rare pediatric disease designations for its experimental therapy PGN-EDO51 for certain Duchenne muscular dystrophy patients.

PGN-EDO51 received the designations to treat the rare genetic disorder, which causes progressive muscle degeneration and weakness, in patients whose mutations are amenable to an exon 51 skipping approach.

The FDA's orphan designation aims to advance the evaluation and development of drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for seven years of market exclusivity after approval, among other benefits.

The rare pediatric disease status is given by the FDA to drugs for serious or life-threatening disease in individuals aged from birth to 18 years and the regulator may award priority review vouchers to sponsors whose drugs received the status.

PepGen said it is assessing the experimental therapy for treating Duchenne muscular dystrophy in the ongoing CONNECT1 Phase 2 trial, and expects to start enrolling patients in the CONNECT2 Phase 2 trial later in 2024.

Write to Zaeem Shoaib at zaeem.shoaib@wsj.com

(END) Dow Jones Newswires

March 13, 2024 07:42 ET (11:42 GMT)

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