Vyjuvek wins UK approval for DEB patients with COL7A1 mutations
Patients can self-administer gene therapy gel at home
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Authorities in the U.K. have approved Krystal Biotech‘s gene therapy gel, Vyjuvek (beremagene geperpavec-svdt), to treat wounds in people with dystrophic epidermolysis bullosa (DEB) who have disease-causing mutations in the COL7A1 gene.
The approval from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) covers the use of Vyjuvek from birth onward, according to a company press release.
“We are delighted that Vyjuvek is now approved in the United Kingdom as the first and only corrective therapy for the treatment of DEB,” said Suma Krishnan, president of research and development at Krystal Biotech.
Vyjuvek met the U.K.’s criteria for orphan designation, which provides incentives to developers of medicines targeting rare diseases. As a result, the therapy will receive up to 12 years of market exclusivity in the U.K.
The approval follows similar decisions in the U.S., the European Union, and Japan. As in those regions, the approval includes flexible administration options, meaning Vyjuvek may be administered by healthcare professionals or at home by patients or caregivers who have received appropriate training.
Reimbursement procedures being worked out
Krystal said the timing of Vyjuvek’s U.K. launch is pending completion of reimbursement procedures, which are underway.
“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,” said Laurent Goux, executive vice president and head of international at Krystal Biotech. “We are now focused on close collaboration with relevant authorities to support broad and rapid access across the United Kingdom.”
DEB is a type of epidermolysis bullosa that’s caused mainly by mutations in the COL7A1 gene, which codes for a protein called type VII collagen that helps hold the layers of skin together. A deficiency of this protein causes the skin to be abnormally fragile, resulting in blisters and widespread wounds that heal slowly.
Vyjuvek is designed to deliver a working version of the COL7A1 gene to skin cells, allowing them to produce their own type VII collagen to help with wound healing. The treatment is delivered as a gel applied to wounds.
Vyjuvek’s approvals were based primarily on data from the Phase 1/2 GEM-1 clinical trial (NCT03536143) and the Phase 3 GEM-3 study (NCT04491604), which demonstrated that the therapy increased type VII collagen levels and promoted durable wound closure.
The U.K. approval was also supported by data from an open-label extension study (NCT04917874) that tracked long-term outcomes, as well as data from real-world studies in other countries.
“This positive decision by the MHRA, our fourth approval following the United States, European Union, and Japan, reflects both the strength of our clinical dataset and our commitment to maximizing access to Vyjuvek worldwide,” Krishnan said.
