Krystal Biotech, the biopharmaceutical firm that creates gene-based therapies for rare diseases. It announced positive results from its ongoing phase III trial on a possible treatment for dystrophic epidermolysis bullosa (dystrophic BE).

It is the first topical, non-invasive and redosable gene currently in development for rare diseases.

The results of its GEM-3 trial in beremagene geperpavec (B-VEC), also known as VYJUVEK. They demonstrated statistical significance in their ability to promote complete wound healing in six months compared to a placebo. VYJUVEK is reportedly the first topical, non-invasive, redosable gene currently in development for rare diseases.

It is also said to be the only gene-based approach to treating dystrophic EB.

The GEM-3 trial is a double-blind, randomized, intrapatient study that evaluates the safety and efficacy of the drug. For the trial, 31 patients aged one year to 44 years were enrolled at three sites. In each patient, some wounds received VYJUVEK topically, while others received a placebo. Wounds were dosed once a week and observed until closed.

At the end of six months, the wounds were evaluated to determine the primary outcome of complete wound healing. Whereas the secondary endpoints were complete wound healing at three months and mean change in pain severity. Measured by a FLACC-R (face, legs, activity, crying, comforting) scale or visual analog scale (VAS).

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VYJUVEK works by delivering two copies of the COL7A1 gene

VYJUVEK works by delivering two copies of the COL7A1 gene when it comes into contact with DEB wounds, operating at the molecular level. Unlike the current standard of care. If positive results continue, it could be the first topical drug that can address the root cause of the disease itself.

At the time of writing this article, the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) granted VYJUVEK an orphan drug designation for DEB. It also has a fast track designation and a rare pediatric designation from the FDA.

“With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first treatment to patients with dystrophic EB and their families who are in desperate need of it. Dystrophic epidermolysis bullosa is known as ‘the worst disease you have ever heard of’ due to the incredibly devastating reality faced by patients with this genetic condition, and we are delighted to announce the positive results of our landmark GEM-3 trial of VYJUVEK, “said Suma Krishnan, founder and COO of Krystal, in a statement.

The latest outcome is the foundation for Krystal’s plan to file a Biologics License Application (BLA) with the FDA in the first six months of 2022.

You will then submit a Marketing Authorization Application (MAA) in Europe after this is done. Krystal is also considering bringing VYJUVEK to Japan, among other countries.

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