Krystal Biotech’s gene therapy candidate KB103 obtained an orphan drug designation from the US Food and Drug Administration. IND application coming next year.
It’s been a good week so far for Krystal Biotech (NASDAQ:KRYS) as the market reacted favorably to the company getting an orphan drug designation for their dystrophic epidermolysis bullosa candidate.
Krystal Biotech enjoyed a 4.60 percent bump to its stock in early trading on Tuesday (November 7) after the US Food and Drug Administration (FDA) granted the company an orphan drug designation for its lead candidate KB103. At Tuesday’s, Krystal Biotech’s stock price had increased 1.32 percent overall to $10 on the nose.
KB103 is a gene therapy treatment for dystrophic epidermolysis bullosa, a genetic rare condition that causes the skin to be fragile and to blister easily, according to the National Institute of Health’s Genetics Home Reference. This condition is caused by a mutation in the COL7A1 gene.
IND application for KB103 coming next year
“In December 2016, we received the designation of ‘rare pediatric disease’ for KB103 that will qualify us to receive a Rare Pediatric Priority Review Voucher upon approval of KB103,” Suma M. Krishnan, founder, and CEO of Krystal Biotech said in the press release.
Krishnan added the company will file an investigational new drug (IND) application with the FDA in the first quarter of 2018.
“We are excited by the results of the pharmacology data in the diseased hypomorphic animal model for DEB that we submitted to the Office of Orphan Product Development (OOPD),” Krishnan said.
Krystal Biotech entered the stock market on September 21, with an initial public offering of 4,554,000 shares of common stock at a $10.00 per share price. The company reported gross proceeds from the offering worth $45,540,000. Since its launch, shares of Krystal Biotech have decreased 6.02 percent.
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Securities Disclosure: I, Bryan Mc Govern, hold no investment interest in any of the companies mentioned.