The European Medicines Agency’s (EMA) human medicines committee has recommended Krystal Biotech’s Vyjuvek (beremagene geperpavec) to treat patients with the ultra-rare genetic blistering disease dystrophic epidermolysis bullosa (DEB).

The Committee for Medicinal Products for Human Use (CHMP) has recommended that the topical gene therapy be approved to treat wounds in DEB patients of all ages in either a healthcare setting or at home.

Usually present at birth, DEB is caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene and is characterised by skin fragility, blisters, small white bumps and scarring.

Most patients receive supportive and palliative care, and there is currently only one treatment authorised for this condition, but it is not disease-modifying.

Vyjuvek, applied to patients’ wounds once a week in small droplets, delivers new COL7A1 genes directly to skin wounds to promote healing.

The CHMP’s decision on the therapy was based on positive data from a controlled trial in 31 patients with a mean age of 17 years, who received Vyjutek and placebo gel separately on two comparable wounds.

Results showed that Vyjuvek significantly improved complete wound healing in 71% of wounds at three months and in 67% at six months, compared to 20% and 22%, respectively, in wounds treated with placebo.

Suma Krishnan, president of research and development at Krystal Biotech, said: “The CHMP’s support for a broad label, including treatment of patients from birth and the option of patient or caregiver administration at home, are also fantastic outcomes for the DEB patients we aim to serve, broadening access and reducing barriers to starting on and staying on therapy.”

The CHMP’s review of Vyjuvek was supported through the EMA’s PRIority MEdicines scheme, which provides enhanced scientific and regulatory support to medicines that have a particular potential to address unmet medical needs.