U.S. FDA approves Krystal Biotech's skin disorder gene therapy (updated)

Krystal Biotech

19/05/2023 19:03

(Adds background, details from FDA statement)
       May 19 (Reuters) - The U.S. Food and Drug Administration
on Friday approved Krystal Biotech Inc's  KRYS.O  topical gene
therapy for patients with a genetic skin disorder, making it the
first-of-its kind treatment for epidermolysis bullosa. 
        The therapy, Vyjuvek, has been approved for the
treatment of dystrophic epidermolysis bullosa, a rare and severe
genetic disease that usually presents at birth. 
        Patients with the disease suffer from open wounds,
causing skin infections and are at an increased risk of vision
loss, scarring and even lead to skin cancer that can be fatal.
Most affected patients rarely survive beyond 30 years of age.
        The company estimates around 9,000-10,000 people suffer
from the skin disorder globally, including a U.S. population of
about 3,000 bearing the brunt of the disorder's recessive and
the dominant forms. 

 (Reporting by Pratik Jain in Bengaluru; Editing by Shailesh
Kuber)
 ((Pratik.Jain@thomsonreuters.com;))