Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD). 

AATD is caused by mutations in the SERPINA1 gene that lead to decreased levels and/or decreased functionality of alpha-1 antitrypsin protein. The protein is primarily produced in the liver and secreted into the bloodstream, where it acts as a circulating serine protease inhibitor whose principal substrate is neutrophil elastase in the lungs. Over time, the deficiency can lead to progressive enzymatic destruction of the lung tissue, ultimately causing life-threatening pulmonary impairment and severe respiratory insufficiency. In severe cases, current disease management includes intravenous augmentation therapy which requires weekly infusions, the clinical benefit of which remains to be established. 

KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD. 

"This important designation is a milestone in the advancement of KB408, and this decision by the FDA underscores the need for potential new treatment options for patients with AATD," said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. "We are encouraged by the profile of KB408 in preclinical studies to date and look forward to dosing patients once we receive clearance from the FDA."  

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Meg Dodge

Krystal Biotech

mdodge@krystalbio.com