FDA Rare Pediatric Disease designation for BPGbio

The US Food and Drug Administration (FDA) has granted a Rare Pediatric disease designation for BPM31510T, an investigational treatment for epidermolysis (EB) from BPGbio, a biology-first, AI-powered, clinical stage biopharma focused on mitochondrial biology and protein homeostasis.

BPGbio noted that this designation coincides with EB Awareness Week, observed annually from October 25-31, highlighting the urgent need for innovative EB treatments.

EB, a rare genetic connective tissue disorder, affects roughly one in 20,000 children in the USA, leaving them with delicate skin prone to blistering, chronic wounds, scarring, and increased cancer risks. Severe cases of EB can result in life-threatening complications, including an aggressive form of squamous cell carcinoma (SCC). Currently, there is no cure and treatment options are limited, the company pointed out.