FDA Grants Approval to First Ever Topical Gene Therapy

Anna Osborne
23 May 2023
Clinical Trials
Gene Editing
Gene Therapy
Viral/Non-Viral Vectors
The FDA has granted approval for the first-ever topical gene therapy, Vyjuvek designed to treat wounds in patients with dystrophic epidermolysis bullosa (DEB).

Vyjuvek is a genetically engineered herpes simplex virus type 1 vector-based gene therapy and becomes the first gene therapy for DEB to earn FDA approval.

The therapy will be mixed into a non-active gel before being applied topically to the wounds of patients with DEB with mutations in the collagen type VII alpha 1 chain (COL7A1) gene, over 6 months of age, in droplets once a week.

DEB is a genetic disorder caused by mutations in the COL7A1 gene which encodes for a type of collagen. The resulting collagen deficiency means the patient’s skin layers can separate leaving painful and debilitating blisters and wounds. Severe and widespread blistering can also lead to serious medical complications, including vision loss.

Managing DEB wounds has been an ongoing challenge, and until now, treatment options have been limited to wound care, pain management, and preventive measures.

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The newly approved Vyjuvek works by delivering COL7A1 gene copies directly to the affected skin cells, promoting wound healing and enhancing the integrity of the skin. During its production, Vyjuvek’s ability to replicate in normal cells is eliminated.

“Today’s action demonstrates the FDA’s ongoing commitment to supporting the development and evaluation of new treatments that address unmet needs for rare diseases or conditions,” said Peter Marks, Director of the FDA’s Center for Biologics Evaluation and Research.

Extensive clinical trials have demonstrated the therapy’s safety and efficacy, with notable improvements in wound closure and reduction of pain observed in DEB patients.

The FDA’s thorough review process assessed the therapy’s benefits, potential risks, and overall impact on patient well-being, leading to its approval as a viable treatment option.

As this innovative therapy becomes available to patients, ongoing research and continued monitoring will be crucial to further assess its long-term effects and optimize treatment protocols.

Collaboration between medical professionals, researchers, and regulatory bodies will play a vital role in ensuring the therapy’s continued success and expanding its accessibility to those in need.

Source: FDA Press Release

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