Posted by Michael Wonder on 10 Sep 2021
Decibel Therapeutics receives orphan drug and rare paediatric disease designations for DB-OTO for the treatment of otoferlin related congenital hearing loss
9 September 2021 - Decibel Therapeutics today announced that the U.S. FDA has granted both orphan drug designation and rare paediatric disease designation for the company’s lead gene therapy product candidate, DB-OTO, for the treatment of patients with otoferlin related congenital hearing loss.
DB-OTO is a dual-vector adeno-associated virus investigational gene therapy product candidate designed to restore hearing to individuals with profound, congenital hearing loss caused by mutations in the otoferlin gene.
