European Commission Grants Orphan Drug Designatipon For AK-OTOF

Release Date: 23-Sep-2021

European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. Akouos, Inc. a precision genetic medicine company has got a positive response for orphan drug designation for AK-OTOF. Around, 20,000 individuals are affected by Otoferlin Gene-Mediated Hearing Loss disease in the U.S and Europe.


AK-OTOF is aimed to cure the OTOF-mediated hearing loss through delivery of a transgene using a dual vector technology which results in expression of normal, functional otoferlin protein in the affected cells, namely inner hair cells, in the cochlea.


Orphan designation by the EMA is designed to encourage the development of new treatments for life-threatening or chronically debilitating conditions affecting no more than five in 10,000 people in the European Union. Medicines that meet the criteria for orphan designation potentially qualify for several incentives, including 10-year market exclusivity, reduced fees for regulatory activities and protocol assistance.

Need custom market research solution? We can help you with that too.