BOSTON - PepGen Inc. (NASDAQ:PEPG), a biotechnology firm focused on developing oligonucleotide therapies for severe neuromuscular and neurological diseases, has received orphan drug and rare pediatric disease designations from the U.S. Food and Drug Administration (FDA) for its investigational drug PGN-EDO51, aimed at treating Duchenne muscular dystrophy (DMD).
The FDA's orphan drug designation is intended to facilitate drug development for rare diseases, typically those affecting fewer than 200,000 people in the United States. This status provides PepGen with various development incentives, including tax credits for clinical trials, fee waivers, and seven years of market exclusivity upon approval.
Similarly, the rare pediatric disease designation targets serious conditions affecting children from birth to 18 years, potentially granting a priority review voucher upon approval of the drug. This voucher can expedite the review process for another product from the company.
PGN-EDO51, PepGen's lead clinical candidate, employs the company's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to target the root cause of DMD, a genetic disorder characterized by progressive muscle degeneration. The therapy aims to restore the production of a functional, albeit truncated, dystrophin protein by skipping exon 51, which could benefit approximately 13% of DMD patients.
PepGen's President and CEO, James McArthur, Ph.D., emphasized the importance of these designations, highlighting the ongoing CONNECT1 Phase 2 trial and the anticipation of preliminary data this year. The company also plans to initiate the CONNECT2 Phase 2 trial later this year.
In preclinical studies, PGN-EDO51 demonstrated promising results, showing greater levels of exon-skipping compared to other candidates. The Phase 1 Healthy Volunteer study indicated six times higher mean exon 51 skipping than the naked oligonucleotide.
DMD is one of the most common rare genetic diseases, with an incidence rate of about one in every 3,500 to 5,000 male births. Despite existing treatments, the disease remains fatal by young adulthood due to limited dystrophin production.
The information in this article is based on a press release statement from PepGen Inc.
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