BERG announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the company's lead product candidate, BPM 31510 (ubidecarenone), for the treatment of patients with Epidermolysis Bullosa (EB), a rare, pervasive and debilitating connective tissue disorder for which there is currently no FDA-approved treatment or cure.
"This recognition by the FDA for topical BPM 31510 is a major milestone that will support ongoing clinical development of our EB program," said Niven R. Narain, BERG Co-founder, President and Chief Executive Officer. "We are committed to the advancement of this asset and look forward to assessing how BPM 31510 may be able to make a difference in the lives of the patients and families dealing with such a life-altering diagnosis."
Research indicates that one in every 20,000 children in the U.S. is born with the EB, which is characterized by extremely fragile skin causing painful blisters, debilitating ruptures and wounds that are prone to infection. The disease significantly diminishes quality of life for those affected, and often leads to a potentially serious form of skin cancer, squamous cell carcinoma that, in some cases, can be lethal for patients before they reach the age of 30.
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In June 2016, an Investigator Sponsored Phase I Study for topical BPM 31510 for the treatment of EB was initiated at the University of Miami Department of Dermatology and Cutaneous Surgery. This study is investigating the safety, efficacy and pharmacokinetics of BPM 31510 in all patient sub-types of EB.
The FDA's orphan-drug designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Orphan-drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemption and seven-year marketing exclusivity after FDA marketing approval is received.
This is the second orphan-drug designation granted to BPM 31510 this year. In January, the FDA granted orphan-drug designation to the intravenous formulation of BPM 31510 for the treatment of patients with pancreatic cancer, a program that is currently in Phase 2 clinical development.
Cancer cells alter metabolism to generate energy from non-mitochondrial pathways to support uncontrolled growth. This allows the cancer cells to escape molecular mechanisms controlling cell death. 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.