Krystal Biotech has received the US Food and Drug Administration (FDA) approval for Vyjuvek (beremagene geperpavec-svdt) to treat dystrophic epidermolysis bullosa (DEB) in patients aged six months and above.
DEB is a serious rare genetic disease that affects the skin and mucosal tissues, where patients suffer from open wounds, which causes recurrent skin infections and fibrosis.
It is caused by one or more mutations in the COL7A1 gene, which is responsible for the production of functional type VII collagen (COL7) protein.
Vyjuvek is a genetically modified herpes-simplex virus, engineered in a laboratory, to deliver normal copies of the COL7A1 gene to enable wound healing.
It is the first redosable gene therapy, and the first and only FDA-approved treatment for both recessive and dominant types of DEB, said the biotechnology company.
Krystal Biotech chairman and chief executive officer Krish S Krishnan 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 our commitment to developing and commercialising novel therapies for patients with rare diseases.”
The FDA approval is supported by two clinical studies, the GEM-1/2 and GEM-3 trials.
In the GEM-1/2 study, topical applications of Vyjuvek resulted in durable wound closure, full-length cutaneous COL7 expression, and anchoring fibril assembly with minimal adverse events.
The GEM-3 trial met both its primary endpoint of complete wound healing at six months and its key secondary endpoint of complete wound healing at three months.
The drug was well tolerated with no reported drug-related serious adverse events, or discontinuations due to treatment-related events.
Krystal Biotech intends to commercialise the drug in the US in the third quarter of this year, with plans to commence the promotion immediately.
Furthermore, the biotechnology company has developed Krystal Connect, a personalised support programme to address the needs of patients, caregivers, and families.
Krystal Biotech research and development president Suma Krishnan said: “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.”
