FDA grants orphan drug designation to inhaled therapy for alpha-1 antitrypsin deficiency

The FDA granted orphan drug designation for KB408 for the treatment of alpha-1 antitrypsin deficiency, according to a press release from Krystal Biotech.

Alpha-1 antitrypsin deficiency (AATD) is caused by mutations in the SERPINA1 gene that result in lower levels or functionality of the alpha-1 antitrypsin protein. Over time, the deficiency can lead to destruction of lung tissue and can be life-threatening.

KB408 (Krystal Biotech) is an inhaled treatment consisting of a replication-defective, nonintegrating HSV-1-based vector that delivers two copies of the SERPINA1 transgene.

“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,” Suma Krishnan, president of R&D at Krystal Biotech, said in the press release. “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.”

Orphan drug designation is given to treatments that address rare diseases affecting fewer than 200,000 people in the United States. The designation provides assistance in developing drugs, tax credits, exemptions from FDA fees and 7 years of marketing exclusivity.