Vyjuvek (beremagene-geperpavec-svdt) is a topical vector-based gene therapy that may be used to treat wounds in patients aged 6 months of age and older with dystrophic epidermolysis bullosa (DEB). It corrects the underlying skin defect of DEB by restoring functional copies of the COL7A1 gene to express COL7 and reassembling the anchoring fibrils that bind the dermis to the epidermis.

DEB is a serious, rare, genetic disorder that is one of four inherited forms of Epidermolysis Bullosa. It is caused by one or more mutations in the COL7A1 gene, resulting in the lack of production of functional type VII collagen (COL7) protein. The COL7 protein forms anchoring fibrils necessary to bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). When there are no anchoring fibrils, the skin becomes extremely fragile, with minor friction or trauma (such as putting on a shirt) causing blisters and tears. Areas of the body primarily affected include the hands, feet, knees, and elbows.

Symptoms of DEB include open wounds, which lead to recurrent skin infections and fibrosis that can cause fusion of fingers and toes, and an increased risk of developing an aggressive form of squamous cell carcinoma (SCC).

Vyjuvek was approved on May 19, 2023, and is the first treatment for DEB to be approved by the FDA.

There are no contraindications to the use of Vyjuvek.

Vyjuvek is a topical gel that is applied by a healthcare provider to the wounds of patients with DEB once a week, either in the home or a healthcare setting. Once the gel is mixed with the biological suspension, the gel is dotted evenly over the wound in a grid-like pattern (1cm by 1cm apart) and then covered with a dressing.

The weekly dose that can be applied depends on the age of the patient.

• 6 months to <3 years: 1.6 x 10⁹ PFU or 0.8mL of reconstituted gel
• 3 years and older: 3.2 x 10⁹  PFU or 1.6mL of reconstituted gel.

The amount needed per wound depends on the size of the wound.

Vyjuvek can be re-dosed weekly as needed.

Vyjuvek is well tolerated and common side effects include itching, chills, redness, rash, cough, and runny nose. In clinical trials, there were no drug-related serious adverse events or discontinuations due to treatment-related events.

To report suspected adverse reactions, contact Krystal Biotech, Inc. at 1-844-557-9782 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch.