EMA has recommended granting a marketing authorisation in the European Union (EU) for Vyjuvek (beremagene geperpavec) to treat wounds in patients of all ages with dystrophic epidermolysis bullosa (DEB).
Dystrophic epidermolysis bullosa is a serious, ultra-rare genetic blistering disease caused by mutations in the collagen type VII alpha 1 chain (COL7A1) gene. This gene is involved in the production of type VII collagen (COL7), a protein crucial in supporting and structuring the layers of the skin. DEB is characterised by skin fragility, blister formation, small white bumps (milia) and scarring, and is usually present at birth. Blisters and erosions may also develop in the mouth, oesophagus, rectum, genitourinary system and eyes, or other mucous tissue. Healing of blisters and erosions can result in debilitating scarring and other serious conditions. Recessive DEB, the most severe form of DEB, can lead to the loss of fingernails and toenails, joint deformities, vision loss and unusually aggressive squamous cell carcinoma (a type of skin cancer).
There is currently one treatment authorised for this condition, which treats the symptoms but is not disease-modifying. Most patients receive supportive and palliative (relieving symptoms) care, including pain medication and lifestyle changes.
Vyjuvek consists of a genetically modified herpes-simplex virus which delivers the normal COL7A1 gene to the wounds. It is applied topically to the individual wounds once a week in small droplets.
The recommendation is based on the results of a randomised controlled trial in 31 patients with a mean age of 17 years, including 19 paediatric patients (age 1 to < 17 years) and three patients less than three years old. Two comparable wounds in each subject were selected and randomised to receive either cutaneous application of Vyjutek or placebo (gel only) weekly for 26 weeks. Complete wound healing was defined as 100% wound closure from the exact wound area selected at baseline.
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% in wounds treated with placebo.
The most common side effects are cough, runny nose, skin and subcutaneous tissue disorders (pruritus, erythema and rash), and chills.
Vyjuvek was supported through EMA's PRIority MEdicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients' unmet medical needs.
In its overall assessment of the available data, the Committee for Advanced Therapies (CAT), EMA's expert committee for cell- and gene-based medicines, found that the benefits of Vyjuvek outweighed the possible risks in patients with DEB. The CHMP, EMA’s human medicines committee, agreed with the CAT’s assessment and positive opinion, and recommended approval of this medicine.
The opinion adopted by the CHMP is an intermediary step on Vyjuvek’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role or use of this medicine in the context of the national health system of that country.
Notes: