Regulatory Krystal Biotech Gains FDA Green Light For Skin-Disorder Gene...

Krystal Biotech Gains FDA Green Light For Skin-Disorder Gene Therapy


Vyjuvek, a medication developed by Krystal Biotech, has become the first therapy for a rare and debilitating skin disease to be authorized by the FDA.

The disease, known as dystrophic epidermolysis bullosa (DEB), is characterized by very fragile skin that tears and blisters with the slightest bit of friction. This results in open wounds that are susceptible to skin diseases and fibrosis. Patients who suffer from DEB also have a higher chance of developing an aggressive form of skin cancer.

Vyjuvek is not only the first drug that can treat this condition, but it is also the first topical gene therapy that has been approved by the appropriate agencies.

Priority evaluation was given to Vyjuvek by the FDA, and the drug was given the go-ahead barely nine months after Krystal submitted its application for approval. Moreover, Vyjuvek is an orphan drug, which could grant it marketing exclusivity for 7 years.

“With the FDA approval of Vyjuvek, the DEB population has reached a monumental milestone in the treatment of this horrible disorder. Our hopes have now been realized for a safe and effective treatment for one of the most devastating symptoms of the disorder,” stated Brett Kopelan, executive director of debra of America, a national advocacy team that provides free programs to people with DEB.

There are two forms of DEB, referred to as recessive and dominant. According to the FDA, the dominant form of the illness is often less severe, but the recessive kind is more incapacitating and has the potential to result in deformity, loss of vision, and other potentially fatal problems.

The condition and the injuries it causes are brought on by a mutation (or mutations) in the COL7A1 gene. This gene is crucial for the production of the protein that binds the inner layer of skin to the outer layer. If there is a defect in the gene, the layers of the skin might readily peel off.

This is when Vyjuvek enters the picture. When directly administered to DEB wounds, the genetically engineered herpes simplex type 1 virus included in the external gel causes copies of the COL7A1 gene to be delivered to the affected tissue. According to the FDA’s statement, a clinical trial involving 31 patients found that 65% of wounds treated with Vyjuvek closed fully, whereas just 26% of wounds treated with placebo did so.

Patients older than 6 months are eligible for approval under this provision. In a statement released by the company, the CEO, Krish Krishnan, noted that receiving regulatory approval brings about an entirely novel approach to treating genetic diseases and represents a significant achievement for DEB patients and their loved ones.

Krystal Biohtech estimates that there are between 9,000 and 10,000 individuals with dystrophic epidermolysis bullosa worldwide, with roughly 3,000 of them living in the U.S. Vyjuvek is Krystal’s first FDA-approved product, and it should hit shelves in the third quarter of 2023. 

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