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Krystal Biotech skin disease therapy gets US FDA's orphan drug designation


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Krystal Biotech skin disease therapy gets US FDA's orphan drug designation

Krystal Biotech Inc. said it received the U.S. Food and Drug Administration's orphan drug designation for its potential skin disease drug, KB105.

The drug is in preclinical development for treating patients with transglutaminase 1, or TGM-1, deficient autosomal recessive congenital ichthyosis, or ARCI.

TGM-1 is an enzyme that prevents dehydration and protects the skin from unwanted toxins, and its deficiency results in ARCI.

ARCI is characterized by the formation of scales, which are plate-like, often of a dark color and cover the whole body and can lead to death among infants.

The Pittsburgh-based company expects to file an investigational new drug application for KB105 in the fourth quarter of 2018.

Krystal Biotech had already received the FDA's orphan drug designation for its lead product candidate, KB103. That drug is expected to treat dystrophic epidermolysis bullosa, an incurable, often fatal skin blistering condition caused by a lack of collagen in the skin.