FDA approves first topical gene therapy to treat wounds in patients with dystrophic epidermolysis bullosa


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Today, the U.S. Food and Drug Administration approved Vijuvec, a herpes simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) aged 6 months and older bottom. Mutations in the type VII collagen alpha 1 chain (COL7A1) gene.

“Vijuvec is the first FDA-approved gene therapy treatment for DEB, a rare and serious genetic skin disorder,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Today’s action demonstrates the FDA’s continued commitment to supporting the development and evaluation of new treatments that address unmet needs for rare diseases and conditions.”

DEB is a genetic disease that affects the connective tissue of the skin and nails and is caused by mutations in the COL7A1 gene. This gene encodes collagen VII (COL7), an essential protein that helps strengthen and stabilize the outer and middle layers of the skin. Deficiency of COL7A1 can cause layers of the skin to slough off, resulting in painful, debilitating blisters and scars. DEB usually develops at birth and depending on the inheritance pattern he is divided into two main types. They are recessive dystrophic epidermolysis bullosa (RDEB) and dominant dystrophic epidermolysis bullosa (DDEB).

Symptoms vary greatly among affected individuals. Patients with DDEB usually have mild cases and present with blisters, mainly on the hands, feet, knees, and elbows. Cases of RDEB can be painful and debilitating, often extensively blistered, and can lead to vision loss, disfigurement, and other serious complications that can be fatal.

Vyjuvek is a genetically engineered (laboratory-engineered) herpes simplex virus used to deliver a normal copy of the COL7A1 gene to wounds. COL7 molecules form elongated bundles that form anchoring fibrils that hold the epidermis (skin) and dermis together. This is essential for maintaining skin integrity. Vyjuvek has also been modified to eliminate its ability to replicate in normal cells. Vyjuvek is mixed into an excipient (non-active ingredient) gel prior to topical application. Medical professionals say that once a week he applies droplets of Vijubec gel evenly to the patient’s wound.

The safety and efficacy of Vyjuvek were primarily established in a randomized, double-blind, placebo-controlled study in a total of 31 DEB patients, including 30 with RDEB and 1 with DDEB. In this study, each patient identified two of her DEB wounds of equivalent size and was randomized to receive either topical Vijevec or placebo on a weekly basis. Subject ages ranged from 1 to 44 years (mean age 17 years). Efficacy was established by improved wound healing, defined as the difference in the percentage of confirmed complete (100%) wound closure between Vijuvec-treated wounds and placebo-treated wounds at 24 weeks. Sixty-five percent of the wounds treated with Vi┼żbec closed completely, whereas only 26% of his placebo-treated wounds closed completely.

In addition, another clinical study in two young patients with RDEB (ages 6 months and 7 months, respectively) treated with weekly topical Bijuvec showed no new safety findings.

The most common side effects associated with Vijubek included itching, chills, redness, rash, cough, and runny nose.

Patients or caregivers should take the following precautions during treatment with Vijubec:

  • Avoid direct contact with healing wounds (touching or scratching) or wound dressings for approximately 24 hours after application of Vijuvec. In case of accidental exposure, patients and exposed persons should clean the affected area.
  • Wash your hands and wear protective gloves when changing wound dressings.
  • The bandages for the first dressing change after Vijuvec treatment should be disinfected with a virucidal agent such as 70% isopropyl alcohol, 6% hydrogen peroxide, or less than 0.4% ammonium chloride, and the disinfected bandages should be placed in a separate sealed plastic bag. and dispose of it as household waste. Place used dressings and cleaners in a sealed plastic bag and dispose of with household waste.

This application received Orphan Drug and Fast Track designations. Vyjuvek also received Regenerative Medicine Advanced Therapies and Priority Review designations, and a Rare Pediatric Disease Priority Voucher. The FDA’s Pediatric Rare Disease Priority Review Voucher Program is intended to encourage the development of new drugs and biologics to prevent and/or treat rare diseases in children.

Approval for Vyjuvek was granted to Krystal Biotech, Inc.

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