Krystal Biotechs’ KB105 Receives Rare Pediatric Disease Designation from the FDA to Treat Patients with TGM-1-deficient Autosomal Recessive Congenital Ichthyosis

Krystal Biotech (Nasdaq:KRYS), a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to the company’s gene therapy candidate KB105, for the treatment of patients with TGM-1-deficient autosomal recessive congenital ichthyosis.

As quoted in the press release:

The FDA grants RPDD for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The RPDD makes the KB105 program eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of KB105 by the FDA.

“We are motivated by compassion and urgency for the patients, families and caregivers affected by ARCI and other serious, life-threatening conditions with high unmet needs,” said Suma M. Krishnan, founder and chief operating officer of Krystal Biotech. “This designation, combined with our receipt of the FDA’s Orphan Drug Designation for KB105 earlier this month, will help support our efforts to speed up the drug development efforts in this indication and, when successful, enable this new therapeutic candidate to be made available to patients as soon as possible.”

Click here to read the full press release.