BPGbio Gets FDA Rare Pediatric Disease Designation for Epidermolysis Bullosa Treatment

BPGbio has recently achieved a significant milestone with the U.S. Food and Drug Administration (FDA) granting a Rare Pediatric Disease Designation for BPM31510T, an investigational treatment for epidermolysis bullosa (EB). This announcement comes during EB Awareness Week, observed from October 25-31, underscoring the urgent necessity for innovative treatments for this rare genetic disorder.

Epidermolysis bullosa is a severe genetic condition affecting approximately one in 20,000 children in the United States. It is characterized by extremely fragile skin that easily blisters, leading to chronic wounds, scarring, and a heightened risk of cancer. In its most severe forms, EB can result in life-threatening complications, including an aggressive type of skin cancer known as squamous cell carcinoma (SCC). Currently, there is no cure for EB, and treatment options are scarce.

Brett Kopelan, Executive Director of DEBRA of America, the major EB patient advocacy organization in the U.S., expressed the community's enthusiasm for the FDA designation. He emphasized that EB families are hopeful for more effective treatments and appreciate BPGbio's dedication to advancing research in this field.

The FDA's Rare Pediatric Disease Designation is aimed at encouraging the development of treatments for serious pediatric diseases that lack approved therapies. This designation makes BPGbio eligible for a priority review voucher upon BPM31510T’s approval, marking the second such designation for BPGbio's BPM31510 franchise in just two months. The initial designation was awarded for primary CoQ10 deficiency. Additionally, BPM31510T has also received Orphan Drug Designation from the FDA.

Dr. Robert S. Kirsner, Chairman and Harvey Blank Professor of Dermatology at the University of Miami Miller School of Medicine, highlighted the challenges of managing EB, which can lead to painful and persistent blistering wounds and, in severe cases, SCC. He noted that BPM31510T showed potential efficacy and was well-tolerated in its Phase I clinical trial as a topical therapy for EB. This new designation and continued clinical development of BPM31510T bring renewed hope to EB patients and their families.

BPM31510T is designed to support wound healing in EB patients by targeting cellular and mitochondrial processes. It influences key pathways involved in wound healing, such as inflammation, cell proliferation, and tissue remodeling. Successful preclinical and Phase I studies have encouraged BPGbio to partner with DEBRA of America to advance BPM31510T into Phase II/III trials, anticipated to commence in 2025.

Niven R. Narain, Ph.D., President and CEO of BPGbio, remarked on the significance of receiving a second Rare Pediatric Disease Designation, emphasizing the role of their NAi Interrogative Biology Platform. He pointed out that conditions like EB, CoQ10 deficiency, and cancer, despite seeming unrelated, share mitochondrial dysfunction as a root cause. BPGbio is committed to advancing BPM31510 in the field of mitochondrial medicine through strategic partnerships to benefit underserved patients.

BPGbio's lead clinical candidate, BPM31510, comes in various formulations, including topical, intravenous, and oral. BPM31510T has completed a Phase I trial for EB and a Phase II trial for SCC. BPM31510IV is under study in an ongoing Phase IIB trial for glioblastoma multiforme and has completed Phase IIA trials for pancreatic cancer. Additionally, BPM31510IV recently received an FDA Rare Pediatric Disease designation for primary CoQ10 deficiency. The company is also planning a potential pivotal trial targeting multiple CoQ10 deficiency mutations.

Vijay Modur, M.D., Ph.D., Chief Medical Officer at BPGbio, expressed pride in offering hope for children suffering from debilitating wounds and pain. Preliminary evidence from Phase I clinical trials suggests that BPM31510 is well-tolerated and potentially effective in improving wound healing, with further validation expected through Phase II/III trials.

BPGbio, headquartered in the greater Boston area, is at the forefront of a new era in medicine. The company combines biology, multi-modal data, and AI to transform the understanding, diagnosis, and treatment of diseases, making significant strides in the fields of oncology, rare diseases, and neurology.