Krystal Biotech Inc.’s groundbreaking topical gene therapy for people with a hereditary skin condition received approval from the US Food and Drug Administration on Friday, driving company shares up 7% in afternoon trade.
Patients with the uncommon dystrophic epidermolysis bullosa condition experience open sores that can become infected, raise the risk of scarring and skin cancer, and result in eyesight loss. Rarely do most patients live past the age of 30.
“For at least the past 18 months, we have been getting ready for a commercial debut. If at all feasible, we want to give every patient in the US access to care,”Krystal Biotech CEO Krish Krishnan told Reuters.
The treatment is Krystal’s first to be licensed in the US and is for patients with either recessive or dominant variants of the illness who are at least six months old.
According to Krystal Biotech, dystrophic epidermolysis bullosa affects 9,000 to 10,000 people worldwide, with roughly 3,000 cases in the United States.