Release Date: 02-Oct-2021
US FDA has granted both Orphan Drug Designation and Rare Pediatric Disease Designation for the company’s lead gene therapy product candidate, DB-OTO, for the treatment of otoferlin-related congenital hearing loss. DB-OTO is a dual-vector adeno-associated virus (AAV) investigational gene therapy product candidate designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene.
Rare Pediatric Disease Designation is granted by the FDA to encourage development of treatments for serious or life-threatening rare diseases in which the disease manifestations initially affect individuals aged from birth to 18 years.
The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.