For immediate release:

Today, the U.S. Food and Drug Administration approved the gene therapy Vyjuvek, a herpes simplex virus type 1 (HSV-1) gene therapy, to treat wounds in patients 6 months of age or older with dystrophic epidermolysis bullosa (DEB) with a mutation ( Mutations) in the collagen type VII alpha 1 (COL7A1) gene.

“Vyjuvek is the first FDA-approved gene therapy for DEB, a rare and serious genetic skin disorder,” said Peter Marks, MD, director of the FDA’s Center for Biology Evaluation and Research. “Today’s action demonstrates the FDA’s continued commitment to supporting the development and evaluation of new therapies that address unmet needs for diseases or rare conditions.”

DEB is a genetic disorder that affects the connective tissue of the skin and nails and is caused by mutation(s) in the COL7A1 gene. This gene encodes collagen type VII (COL7), an essential protein that helps strengthen and stabilize the outer and middle layers of the skin. When there is a deficiency of COL7A1, the layers of the skin can peel off, causing painful and debilitating blisters and wounds. DEB usually presents itself at birth and is divided into two main types depending on the inheritance pattern: recessive atrophic epidermolysis bullosa (RDEB) and dystrophic autosomal dominant epidermolysis bullosa (DDEB).

Symptoms can vary greatly between affected people. Individuals with DDEB usually have mild cases with blistering that primarily affects the hands, feet, knees, and elbows. RDEB cases can be painful and debilitating, and often involve widespread blistering that can lead to vision loss, disfigurement, and other serious medical complications that may be fatal.

Vyjuvek is a transgenic virus (engineered in a lab) that is used to deliver normal copies of the COL7A1 gene into wounds. COL7 molecules arrange themselves in long, thin bundles that form stabilizing fibers that hold the epidermis (epidermis) and dermis together, which is essential for maintaining the integrity of the skin. Vyjuvek has also been modified to eliminate its ability to multiply in normal cells. Vyjuvek is mixed into an excipient (inactive ingredient) gel prior to topical application. A healthcare professional applies Vyjuvek gel evenly in drops to the patient’s wounds once a week.

The safety and efficacy of Vyjuvek were primarily established in a randomized, double-blind, placebo-controlled study that included a total of 31 subjects with DEB, including 30 subjects with RDEB and one subject with DDEB. In the study, two DEB wounds of similar size were assigned to each patient and randomized to receive either topical administration of Vyjuvek or placebo on a weekly basis. The age of the participants ranged from 1 year to 44 years (mean age 17 years). Effectiveness was determined by improvement in wound healing, defined as the difference in the percentage of confirmed complete wound closure (100%) between wounds treated with Vyjuvek and wounds treated with placebo at 24 weeks. Sixty-five percent of the wounds treated with VijuVic closed completely while only 26 percent of the wound treated with placebo closed completely.

In addition, in a different clinical study, two young patients with RDEB (6 and 7 months of age, respectively) received topical Vyjuvek weekly with no new safety outcomes.

The most common adverse reactions associated with Vyjuvek included itching, hives, flushing, rash, cough, and runny nose.

Patients or caregivers should take the following precautions while being treated with Vyjuvek:

  • Avoid direct contact with treated wounds (eg, touching, scratching) and treated wound dressings for approximately 24 hours after applying Vyjuvek. In the event of accidental exposure, patients and exposed individuals should clean the affected area.
  • Wash your hands and wear protective gloves when changing wound dressings.
  • Disinfect dressings from the first dressing change after treating Vyjuvek with a virucidal agent, such as 70% isopropyl alcohol, 6% hydrogen peroxide, or less than 0.4% ammonium chloride, and dispose of disinfected dressings in a sealed plastic bag in the household waste. Dispose of subsequent used bandages and cleaning agents in a sealed plastic bag and dispose of it in the household waste.

This app has received Orphan Drug and Fast Track designations. Vyjuvek also received Advanced Regenerative Medicine Treatment designations, Priority Review, and Priority Review Voucher for Rare Pediatric Illnesses. The FDA’s Rare Pediatric Disease Priority Review Voucher Program aims to encourage the development of new drugs and biologics to prevent and/or treat rare diseases in children.

Vyjuvek approval has been granted to Krystal Biotech, Inc.

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The Food and Drug Administration, an agency of the U.S. Department of Health and Human Services, protects public health by ensuring the safety, efficacy, and security of human and veterinary medicines, vaccines, and other biological products for human use, and medical devices. The agency is also responsible for the safety and security of our country’s food supply, cosmetics, and nutritional supplements, products that emit electronic radiation, and for regulating tobacco products.