Krystal Biotech gets rare peds disease tag from FDA

Krystal Biotech announced that the FDA has granted a rare pediatric disease designation, or RPDD, to the company’s gene therapy candidate KB105, for the treatment of patients with TGM-1-deficient autosomal recessive congenital ichthyosis. ARCI is associated with neonatal morbidity and neonatal mortality as well as the following manifestations that primarily affect older infants and children: hypohidrosis, ectropion, alopecia, digital contractures, failure to thrive and short stature. The RPDD makes the KB105 program eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of KB105 by the FDA.

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