BERG LLC, a Boston-based biopharmaceutical company that merges biology with technology to map the nature of diseases, announced yesterday that the United States Food and Drug Administration (FDA) has granted orphan-drug designation for its lead product candidate, BPM 31510 (ubidecarenone) intended for the treatment of patients with Epidermolysis Bullosa (EB), a connective tissue disorder.

In June 2016, an Investigator Sponsored Phase one Study for topical BPM 31510 for the treatment of EB was started 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 US FDA orphan-drug designation program offers 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 United States. 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.