Read the full news release here.
SOUTH SAN FRANCISCO, Calif., Nov. 16, 2023 (GLOBE NEWSWIRE) — Epic Bio, a biotechnology company developing therapies to modulate gene expression using compact, non-cutting dCas proteins, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EPI-321 for the treatment of facioscapulohumeral muscular dystrophy (FSHD), the most common form of muscular dystrophy in adults. EPI-321 is an investigational therapy being developed as a potential single-dose treatment to suppress abnormal expression of the DUX4 gene.
Epic plans to initiate a first-in-human, Phase 1/2 clinical study of EPI-321 in the first half of 2024. The multi-center study will be designed to assess the safety, activity, and preliminary efficacy of EPI-321 in individuals with FSHD.
“We are pleased the FDA has recognized the unmet need of those living with FSHD by granting this Orphan Drug Designation, and we believe EPI-321 could serve as an important new therapeutic option for these patients,” said Weston Miller, M.D., chief medical officer of Epic Bio. “We are working diligently to advance EPI-321 toward the clinic, and we look forward to generating meaningful clinical data to inform its future development as a potential new treatment for FSHD.”
The FDA has the authority to grant Orphan Drug Designation to therapies to prevent, diagnose or treat a rare disease or condition, defined as those affecting fewer than 200,000 people in the United States. The designation qualifies drug developers for certain financial benefits, including tax credits, prescription drug user fee waivers, and a potential period of 7 years of market exclusivity should EPI-321 receive marketing approval in the U.S.
What does this mean for patients?
The orphan designation does not mean that EPI-321 is now available to patients. What it does is it helps Epic Bio by reducing costs and making the company more attractive to investors. This is good for patients indirectly, in that it helps the company from a business standpoint. The drug still needs to get through Phase 1/2 and Phase 3 clinical trials, as well as the FDA approval process. This might take around 4 years if all proceeds smoothly. We will be following developments closely!