Regeneron Heads to FDA Seeking Approval for Game-Changing Hearing Loss Gene Therapy
Regeneron is preparing to submit an FDA application for its gene therapy DB-OTO to treat profound genetic hearing loss caused by OTOF gene variants, following promising clinical trial results235.
In the pivotal CHORD trial, 11 out of 12 children treated with DB-OTO showed clinically meaningful hearing improvements; three achieved normal hearing levels and some regained the ability to recognize speech without visual cues1234.
The therapy uses AAV vectors to deliver functional OTOF genes to the inner ear, restoring natural acoustic hearing for most participants, with stable or continued improvement seen up to nearly two years after treatment34.
DB-OTO has received FDA designations for Orphan Drug and Fast Track, supporting an accelerated regulatory review process2.
No therapy-related serious adverse events were reported in the trial, indicating a favorable safety profile4.
Regeneron aims to file its formal FDA submission by the end of 2025, marking a significant milestone for genetic therapies targeting pediatric deafness245.
Sources:
1. https://www.benzinga.com/news/health-care/25/10/48182533/regeneron-gene-therapy-shows-significant-hearing-gains-in-genetic-hearing-loss
2. https://trial.medpath.com/news/813f2e04d132b393/regeneron-s-db-oto-gene-therapy-restores-hearing-in-11-of-12-children-with-genetic-hearing-loss
3. https://www.biopharmadive.com/news/regeneron-gene-therapy-hearing-loss-deafness-decibel-otof/802627/
4. https://www.contemporarypediatrics.com/view/regeneron-s-db-oto-shows-sustained-hearing-recovery-in-phase-1-2-chord-trial