en.Wedoany.com Reported - The gene therapy Vyjuvek (beremagene geperpavec) has recently received a Therapeutic Positioning Report (IPT) from the Spanish Agency for Medicines and Medical Devices (Aemps), providing an important scientific endorsement for its clinical use in Spain. The therapy is indicated for the treatment of dystrophic epidermolysis bullosa, commonly known as butterfly skin disease.
Vyjuvek, developed by the US company Krystal Biotech, was approved for marketing by the European Commission in 2025. In Spain, the drug has not yet secured widespread public funding, but patient associations have been calling for months for uniform access to treatment and demanding public funding from the government. Organizations such as DEBRA Piel de Mariposa (Butterfly Skin Association) have launched public campaigns, condemning Spain for lagging behind other European countries in treatment accessibility. Meanwhile, the Spanish Ministry of Health has maintained active negotiations with the pharmaceutical company to advance public funding, though both sides blame each other for the delays. Currently, the treatment is available through special pathways or regional plans, with Andalusia being the first region in the country to provide the therapy to patients at its own expense until a national pricing agreement is reached.
The Therapeutic Positioning Report provides a comprehensive evaluation of existing clinical evidence. Data show that 67.4% of wounds treated with Vyjuvek achieved complete healing within six months, compared to only 21.6% in the placebo group, an absolute difference of 45.8%, which is statistically significant. At three months, the complete wound closure rate was 70.6% in the treatment group versus 19.7% in the control group. Additionally, nearly half of the treated wounds (49.7%) remained completely closed at both three and six months, compared to just 7.1% in the placebo group. The report notes that some wounds may reopen during follow-up but can be retreated with the drug.
The report compares Vyjuvek with conventional treatment approaches. Traditional therapy primarily relies on wound care, bandaging, pain management, and infection prevention. Vyjuvek is a topical gene therapy that delivers functional copies of the COL7A1 gene directly into wounds, promoting the production of type VII collagen, a protein essential for maintaining skin integrity.
In its comparison with existing alternatives, the report notes that the only previously approved treatment for these wounds was Oleogel-S10 (a birch bark gel), but Aemps assessed its efficacy as "modest" with limited clinical relevance. In contrast, both gene therapies demonstrated clinically and statistically significant effects. The report concludes that Vyjuvek is the "preferred option" for patients with dystrophic epidermolysis bullosa.
The safety assessment was generally favorable. Most adverse events were mild to moderate and associated with topical application. No deaths or discontinuations due to toxicity were reported. Aemps emphasizes the need for long-term monitoring of this gene therapy, particularly regarding potential immune risks or skin cancer risks.
The report also supports the possibility of administration at home by trained patients or caregivers, provided the drug is prepared under controlled pharmaceutical conditions and specific safety measures are followed. The publication of this report does not automatically imply public funding for the drug; pricing and reimbursement decisions still require economic evaluation and price negotiations within the National Health System (SNS).
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