Authorisation expands treatment options for UK patients with the rare genetic skin condition dystrophic epidermolysis bullosa.

person applying gel onto hand

Following its recent proposal to update the definition for gene therapy products, the Medicines and Healthcare products Regulatory Agency (MHRA) has approved a new gel-based genetic medicine.

Krystal Biotech’s Vyjuvek (beremagene geperpavec) is designed to treat wounds in patients with dystrophic epidermolysis bullosa (DEB), a rare genetic skin condition.

Vyjuvek works by copying the faulty gene into the cells of the wound to help the skin heal and clinical data has demonstrated its benefit in wound healing. In a study of 31 patients aged 1 to 44 years, at six months, 67 percent of wounds healed completely. In those given placebo, this outcome was observed for 22 percent of wounds.

Julian Beach, MHRA Executive Director of Healthcare Quality and Access, said: “This approval provides a new treatment option for patients living with dystrophic epidermolysis bullosa, a rare genetic condition that can cause fragile skin and recurrent wounds.”

This approval provides a new treatment option for patients living with dystrophic epidermolysis bullosa, a rare genetic condition that can cause fragile skin and recurrent wounds”

Last May, the FDA approved Zevaskyn (prademagene zamikeracel) as the first autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) wounds.

Abeona Therapeutics’ cell sheet-based treatment, also known as pz-cel, enabled wound healing and pain reduction that “lasted for years after a single application”, Dr Jean Tang, Lead Principal Investigator of the Phase I/IIa VIITAL study, said at the time of the approval.

Another notable approval in the space includes last month Regeneron’s Otarmeni becoming the first genetic medicine to be approved by the FDA. The AAV gene therapy is indicated for genetic hearing loss.

Separately, in April, the FDA issued draft guidance designed to improve the safety of genome editing therapies with new recommendations for leveraging next-generation sequencing to evaluate the safety of off-target editing.

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