The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ADVM-053, a preclinical gene therapy candidate being investigated as a potential single‑administration treatment which has the potential to provide sustained levels of the C1 esterase inhibitor (“C1EI”) protein.
“We are pleased to receive the Orphan Drug Designation for ADVM-053 from the FDA,” said Leone Patterson, interim President and CEO of Adverum Biotechnologies, Inc. “We are committed to developing effective treatments for patients living with HAE and the support from the FDA will be invaluable towards this goal. We look forward to submitting our IND application in the fourth quarter.”
Orphan drug designation is granted by the FDA to novel drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. The designation provides incentives for sponsors to develop products for rare diseases, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers. The orphan drug designation also could entitle Adverum Biotechnologies to a seven-year period of marketing exclusivity in the United States for ADVM-053 should the company receive FDA approval for the treatment of HAE for this product candidate.
ADVM-053 (AAVrh.10-C1EI) is designed as a single‑administration treatment with the potential to provide sustained expression of the C1 esterase inhibitor protein to eliminate protein level variability and to prevent breakthrough angioedema attacks. In preclinical studies, a single intravenous administration of ADVM-053 increased C1EI protein expression above therapeutic levels and decreased vascular permeability in a mouse model of HAE.