Krystal Biotech, a Pittsburgh-based gene therapy company, has picked up a key win across the Atlantic, securing UK regulatory clearance for its topical gene therapy VYJUVEK to treat wounds in dystrophic epidermolysis bullosa (DEB). The move expands the drug’s global reach and positions it as the first corrective option for DEB patients in the UK, although when it actually shows up in clinics will still depend on reimbursement talks.
The UK’s Medicines and Healthcare products Regulatory Agency granted marketing authorisation on 15 May to beremagene geperpavec, marketed as VYJUVEK, for treating wounds in DEB patients with COL7A1 mutations, with use authorised from birth. The MHRA said the decision was supported by clinical data demonstrating durable wound closure and noted that it evaluated the product under the International Recognition Procedure.
Krystal confirmed the approval in a company statement and said the marketing authorisation was granted to Krystal Biotech Netherlands B.V. In its announcement, Krystal Biotech described VYJUVEK as the first genetic medicine approved in the United Kingdom for DEB. It highlighted that it can be administered flexibly, either at home or in health care settings. "This latest approval brings VYJUVEK closer to patients in the United Kingdom," Laurent Goux, the company's executive vice president, said in the statement.
How Vyjuvek works and trial data
VYJUVEK is a topical gel designed to deliver functional copies of the COL7A1 gene directly to wounds, to promote healing without altering a patient’s underlying DNA. In its approval notice, the MHRA pointed to Phase 1/2 and Phase 3 data from a study of 31 patients aged 1 to 44, where 67% of VYJUVEK-treated wounds were completely healed at six months, compared with 22% in the placebo group. The agency also noted that the product was cleared under the International Recognition Procedure.
What the U.K. approval means for patients and business
Krystal said VYJUVEK met UK orphan designation criteria and will be listed on the MHRA Orphan Register, a status that can provide up to 12 years of market exclusivity. In its statement, Krystal Biotech framed the UK decision as its fourth regulatory approval for VYJUVEK, following earlier green lights in the United States, the European Union, and Japan. The company added that the timing of any commercial launch in the UK will hinge on reimbursement procedures that are now underway.
Local impact in Pittsburgh
For Pittsburgh’s life science scene, the approval serves as a tangible validation of the city’s growing biotech footprint. Krystal is headquartered in Pittsburgh and has been scaling up its manufacturing and commercial operations in recent years. The Pittsburgh Business Times reported on the UK decision, noting that VYJUVEK revenue featured prominently in Krystal’s early May financial disclosures, which showed a stronger-than-expected first quarter. The full Q1 report was published by Nasdaq.
What's next
For patients, the UK approval clears a major regulatory hurdle between them and a potential corrective therapy. For Krystal, the focus now shifts to the less glamorous but decisive work of pricing and payer negotiations that will determine how quickly VYJUVEK reaches clinics in the United Kingdom. Company representatives said they plan to continue working with regulators and health systems as they move toward a commercial rollout.
