Boston biopharmaceutical company BERG LLC revealed on Wednesday the receipt of the US Food and Drug Administration's (FDA) orphan-drug designation for its lead product candidate, BPM 31510 (ubidecarenone), for the treatment of patients with Epidermolysis Bullosa (EB).
EB is a rare, pervasive and debilitating connective tissue disorder for which there is currently no FDA-approved treatment or cure. The disease diminishes quality of life for those affected and leads to a potentially serious form of skin cancer, squamous cell carcinoma, which can be lethal for patients.
BPM 31510 is a first in class molecule that specifically targets the dysregulated metabolism observed in cancer. BPM 31510, by targeting metabolism in cancer cells, re-engages the mitochondria to generate energy, shifting metabolism to that observed in the normal cell. The effect of BPM 31510 on metabolism results in the reactivation of pathways that detect cell damage, triggering apoptosis or programmed cell death, added the company.
The US FDA's orphan-drug designation is based on an Investigator Sponsored Phase I Study at the University of Miami Department of Dermatology and Cutaneous Surgery for topical BPM 31510 for the treatment of EB . This study is investigating its safety, efficacy and pharmacokinetics in all sub-types of EB.
This is the second orphan-drug designation granted to BPM 31510 this year. In January, the US FDA granted orphan-drug designation to the intravenous formulation of BPM 31510 for the treatment of patients with pancreatic cancer, concluded the company.
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