Krystal Biotech secures FDA approval for redosable topical gene therapy

James Spargo | May 22, 2023 | News story | Medical Communications FDA, Krystal Biotech, Paediatrics, dystrophic epidermolysis bullosa, gene therapy 

US-based biotech company Krystal Biotech has announced that the US Food and Drug Administration (FDA) has approved its topical gene therapy for patients six months and older with dystrophic epidermolysis bullosa (DEB). The FDA also granted Krystal a Rare Paediatric Disease Priority Review Voucher (PRV).

Vyjuvek (beremagene geperpavec-svdt), a novel redosable gene therapy, is designed to address the genetic root cause of DEB by delivering functional copies of the human COL7A1 gene directly to the skin. COL7A1 is responsible for the production of functional COL7 protein which forms the anchoring fibrils necessary to bind the dermis to the epidermis. As DEB patients lack, or have defunct versions of, COL7A1, their skin is extremely fragile. This leads to blistering or tears with even minor trauma, which create open wounds susceptible to skin infections. It can also lead to fibrosis which can cause fusion of fingers and toes and an increased likelihood of aggressive skin cancer.

The FDA’s approval of Vyjuvek is based on two clinical studies: GEM-1/2 and GEM-3. GEM-1/2 was an intra-patient, open label, single centre, randomised, placebo-controlled study which proved that repeat topical applications of Vyjuvek lead to durable wound closure, full-length cutaneous COL7 expression and anchoring fibril assembly.

GEM-3 met both its primary and secondary endpoints: complete wound healing at six months and complete wound healing at three months, respectively.

Suma Krishnan, president of Research and Development at Krystal Biotech, stated: “Data from our GEM-1/2 trial and our GEM-3 trial, published in Nature Medicine and The New England Journal of Medicine, respectively, demonstrated the strength of both studies showing that Vyjuvek safely and effectively improved wound healing. For so many years, all we have been able to offer DEB patients was palliative care, but now, based on the strength of the Company’s clinical trial data, there is a safe and effective FDA approved treatment.”

Krish S Krishnan, chairman and CEO of Krystal Biotech, said: “Today’s landmark approval of Vyjuvek as the first redosable gene therapy ushers in a whole new paradigm to treat genetic diseases and is an important milestone for patients affected by DEB as well as their families and caregivers. We offer our sincere gratitude to DEB patients, caregivers, investigators, US regulators and our employees who made this approval possible. For Krystal, this is a transformative achievement that highlights … Krystal’s capability as a fully-integrated company ready to launch and bring Vyjuvek to patients as quickly as possible and deliver additional transformative medicines to patients as we advance our pipeline.”

James Spargo

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