FDA grants BPGbio rare pediatric disease designation for epidermolysis bullosa treatment
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Key takeaways:
- BPM31510T previously received orphan drug designation.
- Phase 2/ 3 trials are expected to commence in 2025.
The FDA granted rare pediatric disease designation for BPM31510T for the treatment of epidermolysis bullosa, BPGbio announced in a press release.
Coinciding with Epidermolysis Bullosa (EB) Awareness Week, the FDA’s designation of BPM31510T brings a potential treatment for the rare genetic connective tissue disorder that affects about one in 20,000 children in the U.S. EB is characterized by delicate skin with blistering, chronic wounds, scarring and an increased risk for cancer, and severe cases can have life-threatening complications. There is currently no cure for the condition, with limited treatment options.
“We are honored to offer hope for children who suffer from debilitating wounds and pain,” Vijay Modur, MD, PhD, chief medical officer of BPGbio, said in the release. “Preliminary evidence from phase 1 clinical trial suggests BPGbio’s BPM31510 appears to be well-tolerated and potentially efficacious in improving wound healing. We’re looking forward to validating these results further through phase 2/3 trials.”
According to the release, rare pediatric disease designation is reserved for treatments for diseases that lack approved therapies. BPGbio would be eligible for a priority review voucher if BPM31510T for EB is approved. BPM31510T previously received orphan drug designation from the FDA.
BPGbio has partnered with debra of America, the largest advocacy organization for patients with EB in the U.S., to evaluate the investigational treatment in phase 2/3 trials for EB. These trials are expected to begin in 2025.