Pepgen Inc. (NASDAQ: PEPG) has announced that it has received orphan drug and rare pediatric disease designations for PGN-EDO51 from the U.S. Food and Drug Administration (FDA). The FDA granted these designations for the treatment of Duchenne Muscular Dystrophy (DMD) patients whose mutations are amenable to an exon 51 skipping approach.
The orphan drug designation is significant as it provides incentives for the evaluation and development of new treatments for rare diseases. It includes tax credits for qualified clinical trials, FDA user-fee benefits, and seven years of market exclusivity in the United States after approval.
Under the rare pediatric disease program, the FDA may award priority review vouchers to sponsors who have been granted rare pediatric disease designation for their drug. This program is focused on serious or life-threatening diseases that primarily affect individuals aged from birth to 18 years.
Pepgen is currently evaluating PGN-EDO51 for the treatment of DMD in the ongoing Connect1 phase 2 trial. The company expects to begin enrolling patients in the Connect2 phase 2 trial later this year.
James McArthur, Ph.D., President and CEO of Pepgen, emphasized the critical demand for novel and enhanced therapeutic options for people living with DMD, stating that the receipt of these FDA designations for PGN-EDO51 underscores this need.
Pepgen is looking forward to reporting preliminary data from the Connect1 phase 2 trial later this year. These developments mark significant progress in the company's efforts to advance the next generation of oligonucleotide therapies and transform the treatment of severe neuromuscular and neurological diseases.
As a result of these announcements, the company's shares have moved 1.5% on the market, and are now trading at a price of $14.1. Check out the company's full 8-K submission here.
