Adverum Biotechnologies announced that the United States 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 protein.
HAE affects approximately 8,000 individuals in the U.S. This disease is caused by a genetic mutation that results in low levels of C1 esterase inhibitor which can be associated with sudden swelling and edema of respiratory airways, gastrointestinal tract, and extremities.
“We are pleased to receive the Orphan Drug Designation for ADVM-053 from the FDA,” said Leone Patterson, interim president and chief executive officer of Adverum Biotechnologies. “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.