Akouos Receives Orphan Drug & Rare Pediatric Disease Designations 

Akouos, Inc. recently announced the US 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 US 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.”

The Office of Orphan Products Development of FDA grants ODD to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases, or conditions affecting fewer than 200,000 people in the US. The designation affords Akouos the potential for certain benefits, including up to seven years of post-approval market exclusivity, assistance in the drug development process, tax credits for clinical development, and exemptions from certain FDA fees.

Rare pediatric disease designation is granted by FDA to encourage development of treatments for serious or life-threatening rare diseases in which the disease manifestations primarily affect individuals aged from birth to 18 years. Under the Priority Review Voucher program, and subject to FDA approval of AK-OTOF for the treatment of OTOF-mediated hearing loss, Akouos may be eligible to receive one priority review voucher, which could then be redeemed to receive priority review for a subsequent marketing application for a different product or sold or transferred to another sponsor.

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.