Wednesday, September 06, 2023
Krystal Biotech, Inc. (NASDAQ: KRYS), a biotechnology firm specializing in genetic medicine development to address diseases with significant unmet medical needs, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for KB408 as a potential treatment for alpha-1 antitrypsin deficiency (AATD).
AATD is a condition caused by mutations in the SERPINA1 gene, resulting in reduced levels and impaired function of the alpha-1 antitrypsin protein. This protein is primarily produced in the liver and released into the bloodstream, where it plays a critical role in inhibiting neutrophil elastase in the lungs. Over time, AATD can lead to progressive lung tissue damage, ultimately causing life-threatening pulmonary issues and severe respiratory insufficiency. The current management for severe cases involves weekly intravenous augmentation therapy, and its clinical effectiveness is still being investigated.
KB408 is a nebulized formulation of Krystal Biotech's innovative, non-integrating HSV-1-based vector. It is designed to deliver two copies of the SERPINA1 transgene, responsible for producing human alpha-1 antitrypsin protein, addressing the treatment of AATD.
Suma Krishnan, President of Research & Development at Krystal Biotech, Inc., emphasized the significance of this FDA designation for KB408 and the urgent need for new treatment options for AATD patients. The company is optimistic about KB408's performance in preclinical studies and anticipates commencing patient dosing once it receives FDA clearance.
Orphan Drug Designation is granted by the FDA to investigational therapies targeting rare medical diseases or conditions affecting fewer than 200,000 people in the United States. It offers several benefits to drug developers, including assistance throughout the drug development process, tax credits for clinical expenses, exemptions from certain FDA fees, and seven years of marketing exclusivity following approval.