The United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization on Friday to Pittsburgh-based Krystal Biotech, a biotechnology company focused on genetic medicines, for its drug VYJUVEK.
VYJUVEK is the first genetic medicine approved in the United Kingdom for the treatment of wounds in patients with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene, from birth. The drug delivers functional copies of the human COL7A1 gene to provide wound healing and sustained functional type VII collagen protein expression with redosing.
“This latest approval brings VYJUVEK closer to patients in the United Kingdom and advances our vision of providing corrective therapy to as many DEB patients as possible,” Laurent Goux, Krystal Biotech executive vice president and head of international, said. “We are now focused on close collaboration with relevant authorities to support broad and rapid access across the United Kingdom.”
Approval includes flexible administration options, allowing for dosing at home or in a healthcare setting, with the option for administration by patients or caregivers. The drug fulfilled the United Kingdom Orphan Designation criteria and was added to MHRA’s Orphan Register, allowing it to benefit from up to 12 years of market exclusivity.