FDA approves First repeatable drug gene Therapy, $630,000 a Year, for 'most painful diseases'
2023-05-23
On May 19, 2023, the FDA approved Beremagene Geperpavec (B-VEC), a topical gene therapy based on the herpes simplex virus type 1 (HSV-1) developed by Krystal Biotech, under the trade name Vyjuvke. For the treatment of wounds in patients 6 months of age and older with malnourished epidermolysis bullosa (DEB) with COL7A1 gene mutation. Dystrophic Epidermolysis Bullosa (DEB) is a rare genetic skin disease caused by mutation of COL7A1 gene, which codes type VII collagen (C7). The patient is prone to skin bullosa and blood bullosa. These patients are also known as "butterfly babies", describing their skin as fragile as a butterfly that breaks at the touch. It is one of the most painful diseases in the world, and sufferers are prone to infection, skin cancer and even death.