AK-OTOF to treat otoferlin gene-mediated hearing loss in EUSeptember 11, 2021
The European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss, Akouos, Inc announced.
The positive opinion was subsequently adopted by the European Commission. AK-OTOF was previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designa…
Otoferlin gene (OTOF)-mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the OTOF gene. The OTOF gene encodes otoferlin, a protein that enables the inner hair cells of the cochlea to release neurotransmitter vesicles in response to stimulation by sound to activate auditory neurons.
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