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FX.co ★ PepGen Secures FDA's Orphan Drug, Rare Pediatric Disease Designations For PGN-EDO51

PepGen Secures FDA's Orphan Drug, Rare Pediatric Disease Designations For PGN-EDO51

On Wednesday, PepGen Inc., also known as PEPG, revealed that their potential treatment for Duchenne muscular dystrophy (DMD), PGN-EDO51, has been granted both orphan drug and rare pediatric disease statuses by the FDA. The firm suggested that PGN-EDO51 could be particularly effective for patients possessing mutations that can be addressed using the exon 51 skipping method. Currently, PepGen is in the process of evaluating PGN-EDO51 through the CONNECT 1 Phase 2 trial and they plan to initiate patient enrolment for the CONNECT 2 Phase 2 trial later this year.

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