Akouos, Inc (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with hearing loss worldwide, announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company’s application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. The positive opinion was subsequently adopted by the European Commission. AK-OTOF was previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the US Food and Drug Administration (FDA) for this same indication.
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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. Most individuals with OTOF-mediated hearing loss have severe-to-profound sensorineural hearing loss from birth and approximately 20,000 individuals are affected in the United States and Europe. AK-OTOF is designed to treat the underlying cause of OTOF-mediated hearing loss through delivery of a transgene using a dual-vector technology that results in expression of normal, functional otoferlin protein in the affected cells, namely inner hair cells, in the cochlea, according to the company.
“Severe-to-profound sensorineural hearing loss from birth caused by mutations in the OTOF gene is a high unmet need, especially given that there are currently no approved pharmacologic treatment options available,” said Katie Wachtel, MS and vice president of regulatory affairs of Akouos. “The orphan drug designation granted by the European Commission is an important step towards advancing the global development of AK-OTOF, a gene therapy with potential to restore physiologic hearing and provide long-lasting benefit to individuals with OTOF-mediated hearing loss. Along with the ODD and RPDD designations previously granted by FDA, the orphan drug designation for AK-OTOF in the European Union could accelerate our development of AK-OTOF and our progress towards our mission of healthy hearing available to all.”
Orphan drug designation in the European Union is granted by the European Commission based on an opinion issued by the EMA COMP. An orphan drug designation provides a number of benefits, including fee reductions, regulatory assistance, and the possibility to apply for a centralized European Union marketing authorization. Marketing authorization for an orphan drug can lead to a 10-year period of market exclusivity.