Krystal Biotech’s topical gene therapy Vyjuvek in an eyedrop formulation has restored the vision of a 13-year-old boy with the genetic condition dystrophic epidermolysis bullosa that scarred his eyes, according to a case brief published Thursday in The New England Journal of Medicine.
Tag Archive for: dystrophic epidermolysis bullosa
Chiesi Global Rare Diseases gained access to the Filsuvez topical gel in January 2023, when it bought Amryt Pharma. Tuesday’s approval comes nearly two years after an initial rejection by the regulator.
The topical treatment Vyjuvek got the FDA’s greenlight, making it the first redosable gene therapy and the first therapeutic for the rare skin disease dystrophic epidermolysis bullosa.