Akouos Receives Orphan Drug and Rare Pediatric Disease Designations for AK-OTOF for the Treatment of Otoferlin Gene-Mediated Hearing Loss

Akouos, Inc. (NASDAQ: AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. Akouos anticipates that it will submit an investigational new drug application (IND) for AK-OTOF in the first half of 2022.

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.

“There are currently no pharmacologic treatment options for individuals with OTOF-mediated hearing loss, or for any other form of sensorineural hearing loss. The nonclinical data reported to date for AK-OTOF demonstrate durable recovery of auditory function and support future clinical development.” said Jen Wellman, chief operating officer of Akouos. “We believe these are the first Orphan Drug and Rare Pediatric Disease designations granted by FDA for a genetic form of hearing loss, and represent an important milestone for the field of inner ear genetic medicines. The receipt of both designations could help us accelerate development of AK-OTOF, a therapy that we believe has the potential to restore physiologic hearing and provide long-lasting benefits to these individuals and their families.”

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