The U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation for BPGbio, Inc.’s BPM31510T, an investigational treatment for epidermolysis bullosa (EB).
BPM31510T targets cellular and mitochondrial processes that promote wound healing. BPM31510T has completed a phase 1 trial for EB and phase 2 for SCC.
The FDA’s Rare Pediatric Disease Designation is intended for companies developing treatments for pediatric diseases that currently lack approved therapies. This designation provides BPGbio eligibility for a priority review voucher upon BPM31510T’s approval for EB. This designation coincides with EB Awareness Week, which is observed annually from October 25-31.
“EB is difficult to manage clinically and on a day-to-day basis due to fragile skin resulting in painful and persistent blistering wounds that can, in some subtypes, progress into squamous cell carcinoma, an aggressive form of skin cancer, which is the leading cause of mortality in the severe form of EB,” says Robert S. Kirsner, MD, PhD., Chairman and Harvey Blank Professor of Dermatology at the University of Miami Miller School of Medicine and Vice President of the Wound Healing Society, in a news release. “In its Phase I clinical trial, BPM31510T was well-tolerated and demonstrated potential efficacy as an easy-to-use, topical therapy for EB. This designation, and the continued clinical development for BPM31510T, offers new hope to patients suffering from EB and their families.”
“The EB community is thrilled by this designation, as EB patients and families remain hopeful for more effective treatments,” says Brett Kopelan, Executive Director of DEBRA of America. “We deeply appreciate BPGbio’s commitment to advancing research and expanding options for the EB community.”
