Abeona Therapeutics Gets Rare Pediatric Disease Designation

Abeona Therapeutics said early Tuesday that the US Food and Drug Administration has grated a rare pediatric disease designation to its EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa, including recessive dystrophic epidermolysis bullosa, which are life-threatening genetic skin disorders characterized by skin blisters and erosions that cover the body.

With this designation, the FDA may give the company a pediatric priority review voucher if the drug is approved for the pediatric indication. That voucher could then be used by the company for another drug to be given a priority review.

Leave a Comment