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PepGen Announces US FDA Orphan Drug and Rare Pediatric Disease Designations for PGN-EDO51

March 13, 2024

Defeat Duchenne Canada is pleased to share this DMD Community letter from PepGen:


PepGen is pleased to announce that we have received US FDA Orphan Drug and Rare Pediatric Disease Designations for PGN-EDO51 for the treatment of Duchenne muscular dystrophy.

Orphan Drug 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.

In Rare Pediatric Disease Designation the FDA may award priority review vouchers to sponsors who have been granted rare pediatric 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.  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.

PepGen is evaluating PGN-EDO51 for the treatment of DMD in the ongoing CONNECT1 Phase 2 trial in Canada, and expects to begin enrolling patients in the CONNECT2 Phase 2 trial later this year.


The full press release can be accessed below.

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