Adverum Biotechnologies, Inc. (Nasdaq: ADVM) announced on 8/24/18 that the United States (U.S.) 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 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.”

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.

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.