Krystal gets expanded approval for redosable gene therapy

The U.S. FDA approved a label update for Krystal Biotech’s Vyjuvek, expanding the eligible patient population to include dystrophic epidermolysis bullosa (DEB) patients from birth and providing patients and their caregivers the ability to apply Vyjuvek themselves.

Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, was approved in May 2023 for the treatment of wounds in patients 6 months of age and older with the serious, rare skin disease, DEB, with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The topical gene therapy is designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds.

The original approval required that the therapy was administered by a health care professional, either in a health care setting or in the patient's home. Now, in addition to allowing DEB patients and their caregivers to apply Vyjuvek at home on their own, the label update also affords patients greater flexibility in managing wound dressings, allowing application during their standard of care regimen.

The label update is based on real-world data collected since Vyjuvek launched in the U.S., as well as results from the open label extension study published earlier this year.