2023-06-06| Trials & Approvals

Krystal Biotech’s First Topical Gene Therapy Approved by FDA

by Sinead Huang
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Vyjuvek (beremagene geperpavec-svdt) by Krystal Biotech for the treatment of dystrophic epidermolysis bullosa (DEB), a rare skin disorder, was approved by the U.S. Food and Drug Administration (FDA). Krystal Biotech is dedicated to developing gene therapies for rare skin diseases, and Vyjuvek is not only their first FDA-approved drug but also the world’s first non-invasive topical gene therapy. It is also the only gene therapy that can be repeatedly used, marking a significant milestone in the development of gene therapies.

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DEB: Global Pain and Suffering Affecting Thousands

DEB is a severe rare genetic disease that affects the skin and mucosal tissues, which is life-threatening and leaves patients with constant physical scars. Prior to the launch of Vyjuvek, the only available treatment options were palliative care aimed at alleviating pain and symptoms. According to the data from Dystrophic Epidermolysis Research Association of America, approximately 9,000 to 10,000 people worldwide, including around 3,000 in the United States, suffer from this debilitating condition. This highlights the significance of Vyjuvek’s approval and availability in the United States as a crucial breakthrough for DEB patients.

Vyjuvek, bringing New Hope for Patients with DEB

Gene therapy is a treatment approach that aims to improve genetic defects by delivering genes into the body using different vectors. Typically, invasive methods are used to introduce functional vectors into the body, enabling them to replace malfunctioning genes or directly deliver therapeutic genes to treat diseases, and even deactivate problematic genes. In contrast to traditional gene therapies, Vyjuvek adopts a non-invasive, topical application design that can be directly applied to the wounds associated with the disease.

Vyjuvek is a unique gel specifically developed to treat DEB, a condition where functional COL7 protein is not produced due to mutations in the COL7A1 gene. Vyjuvek revitalizes individuals with DEB by restoring the activity of the COL7A1 gene, prompting their skin cells to produce normal COL7 protein. This innovative therapy addresses the fundamental genetic issue of DEB, bringing renewed hope to those affected by the condition.Vyjuvek is authorized for use by healthcare professionals in clinics or at home to treat patients aged six months and above with either dominant or recessive DEB.

A Promising Future for Vyjuvek

Suma Krishnan, President of Research and Development at Krystal Biotech stated, “For so many years, all we have been able to offer DEB patients was palliative care, but now, there is a safe and effective FDA-approved treatment.” 

After the FDA approval, Krystal Biotech estimates that the private placement equity financing will raise approximately $160 million, demonstrating the high expectations placed on Vyjuvek. Krish S Krishnan, Chairman and CEO of Krystal Biotech, Inc. said “These additional funds, together with our existing cash, cash equivalents and investments, should allow us to fund the VYJUVEK launch, future operations, and the advancement of our growing pipeline through the end of 2026. It also allows us to retain the optionality of monetizing the Priority Review Voucher we received in connection with the FDA approval of VYJUVEK at a future date with favorable terms and without any dilution.”

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