Vyjuvek is designed to treat the genetic root cause of DEB
Krystal Biotech (KRYS) announced the U.S. Food and Drug Administration has approved Vyjuvek for the treatment of patients six months of age or older with dystrophic epidermolysis bullosa, or DEB.
DEB is a rare disease that appears at birth or during the first few years of life, and lasts a lifetime. Prognosis is variable, but tends to be serious. Life expectancy is 50 years, and the disease brings with it complications related to infections, nutrition and neoplastic complications.
“Vyjuvek is designed to address the genetic root cause of DEB by delivering functional copies of the human COL7A1 gene to provide wound healing and sustained functional COL7 protein expression with redosing.
Vyjuvek is the first-ever redosable gene therapy and the first and only medicine approved by the FDA for the treatment of DEB, both recessive and dominant, that can be administered by a healthcare professional in either a healthcare professional setting or in the home,” the company stated.
With this approval, the FDA issued the Company a Rare Pediatric Disease Priority Review Voucher, or PRV, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review, the company noted.
“Vyjuvek is expected to be available in the United States in the third quarter of 2023, and the company will begin the promotion of Vyjuvek immediately.
Outside of the US, the European Medicines Agency has granted Vyjuvek orphan drug designation and PRIME – PRIority MEdicines – eligibility for the treatment of DEB.
The Company anticipates starting the official Marketing Authorization Application procedure in the second half of 2023 with a potential approval in 2024.
The Company is also working with the Pharmaceuticals and Medical Devices Agency in Japan to study Vyjuvek and seek approval for potential launch in 2025,” the company added.
KRYS is up 8.7% to $95.00 per share on heavy trading volume.
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