Posted by Michael Wonder on 26 Aug 2018
Krystal Biotechs’ KB105 receives rare paediatric disease designation from the FDA to treat patients with TGM-1-deficient autosomal recessive congenital ichthyosis
23 August 2018 - KB105 is Krystal’s second "off-the-shelf" topical gene therapy candidate to receive rare paediatric disease designation for treating debilitating skin diseases.
Krystal Biotech today announced that the U.S. FDA has granted a rare paediatric disease designation to the company’s gene therapy candidate KB105, for the treatment of patients with TGM-1-deficient autosomal recessive congenital ichthyosis (ARCI).
ARCI is associated with neonatal morbidity (including life threatening dehydration, respiratory problems, and systemic infection) 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.
