Monday, July 10, 2023
Sensorion, a clinical-stage biotechnology company specializing in developing innovative therapies for hearing loss disorders, has announced the submission of its first Clinical Trial Application (CTA) for OTOF-GT to the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom. The company aims to evaluate the safety, tolerability, and efficacy of OTOF-GT through a phase 1/2 clinical trial called Audiogene. The trial will focus on pediatric patients up to 31 months old with otoferlin gene-mediated hearing loss. Prior to the CTA submission, Sensorion conducted extensive preclinical studies to assess the gene therapy's safety and efficacy, successfully manufacturing the gene therapy Drug Product for the upcoming clinical trial.
The OTOF-GT gene therapy program developed by Sensorion utilizes a dual AAV vector to address severe to profound sensorineural prelingual non-syndromic hearing loss caused by OTOF gene mutations. Otoferlin, a crucial protein expressed in the inner hair cells of the cochlea, plays a vital role in signal transmission to the auditory nerve. OTOF-related hearing loss accounts for about 8% of congenital hearing loss cases, affecting an estimated 20,000 individuals in the US and Europe. Notably, OTOF-GT has previously been granted Orphan Drug Designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), as well as Rare Pediatric Disease Designation by the FDA in late 2022.
CEO of Sensorion, Nawal Ouzren, emphasized the significance of this milestone for the OTOF-GT program and Sensorion's broader gene therapy initiatives. The CTA filing marks the first step in a series of planned submissions, with the hope of commencing patient recruitment pending regulatory approval. Sensorion remains committed to collaborating with clinicians, regulatory bodies, and patient groups to address the unmet medical needs in the intricate field of hearing disorders.
Géraldine Honnet, Chief Medical Officer of Sensorion, highlighted the absence of approved drugs for patients with otoferlin gene mutations and the company's goal to revolutionize the standard of care for newborns with OTOF-related conditions. By reducing reliance on cochlear implants, Sensorion aims to significantly enhance the quality of life for these individuals. Honnet expressed excitement about advancing OTOF-GT into clinical development, as gene therapy holds tremendous potential for providing permanent solutions to patients with OTOF mutations and other inner ear diseases.