The Boston-based biopharmaceutical company Berg LLC has announced that their experimental drug candidate BPM 31510 (ubidecarenone) has been awarded Orphan Drug designation by the US Food and Drug Administration (FDA) for the treatment of epidermolysis bullosa. The full article can be read here, at BioSpace.

Epidermolysis bullosa (EB) is an umbrella term for a group of rare inherited skin disorders. Approximately one in every 20,000 people in the US are thought to be born with the EB. The condition is characterised by fragile skin that, when irritated, can produce blisters. People can be affected the blisters on many areas of their body, including inside the mouth, and on the hands and feet. Other symptoms associated with the condition include scarred and/or thickened skin, and thickened nails. There are three main forms of EB: epidermolysis bullosa simplex (EBS), junctional epidermolysis bullosa (JEB), and dystrophic epidermolysis bullosa (DEB). EBS is the most common form, while JEB is the most severe and least common. DEB can be milder, or severe. There is currently no FDA approved cure, and treatments for EB generally focus on managing the symptoms and preventing complications, such as infections.

The drug under development by Berg, BPM 31510, is hoped to eventually provide another treatment option for patients with EB. The drug is being developed in partnership with Debra of America, a non-profit organisation that provides support to the EB community. A study into its safety and effectiveness at treating EB was begun in 2016. The CEO of Ber, Niven R. Narain, has described the FDA’s decision to award the drug Orphan Designation as a “major milestone.” This new status is reserved for drugs intended to treat rare conditions and will grant the developers several benefits to encourage and speed up the drug’s development process. These include a longer period of marketing exclusivity, tax benefits, and prescription drug user fee exemption.

BPM 31510 has also previously been awarded Orphan Drug designation for treating pancreatic cancer. As a cancer drug, it works by changing cell metabolism pathways to trigger the processes that identify cell damage and cause cell death. This is thought to help the body to remove damaged cancer cells. It is currently in Phase 2 clinical trials for this use.

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