Sensorion (FR0012596468 – ALSEN) a clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat, and prevent within the field of hearing loss disorders, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to OTOF-GT, Sensorion’s lead gene therapy program, intended for the treatment of otoferlin gene-mediated hearing loss. Sensorion is on track to file a Clinical Trial Application for OTOF-GT in the first half of 2023.
Sensorion’s OTOF-GT dual vector AAV gene therapy development program aims to restore hearing in people living with otoferlin deficiency. Patients with mutations in OTOF suffer from severe-to-profound sensorineural pre-lingual non syndromic hearing loss. Otoferlin deficiency is responsible for up to 8% of all cases of congenital hearing loss, with around 20,000 people affected in the US and Europe1.
Sensorion has progressed preclinical and clinical development plans for OTOF-GT and is on track to file a Clinical Trial Application (CTA) in the first half of 2023. In September 2022, the Company received a positive opinion on an application for Orphan Drug Designation (ODD) from the European Medicines Agency (EMA). The European Commission issued the decision on October 11, 2022. On November 7, 2022, Sensorion has also been granted Rare Pediatric Disease Designation to OTOF-GT by the US FDA.
“We are really pleased to have received this significant regulatory feedback from the FDA, following the agency’s recent award of Rare Pediatric Disease designation for OTOF-GT. Orphan Drug Designation will support us in advancing our development program, a gene therapy which offers the potential to help patients with a condition for which there are currently no approved curative therapies,” said Géraldine Honnet, Chief Medical Officer of Sensorion. “We are excited to have achieved this milestone and remain highly focused on the development of our most promising candidates to produce life-changing therapies to restore, treat, and prevent hearing loss disorders.”
Orphan Drug Designation is granted by the FDA to encourage development of treatment, diagnosis, or prevention of rare diseases, defined as those affecting fewer than 200,000 people in the US. The designation affords Sensorion 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 for certain FDA fees.
Under Rare Pediatric Disease designation, a sponsor who receives an approval for a drug or biologic for a “rare pediatric disease” may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product or sold to another sponsor for priority review of their marketing application, an opportunity for which there is a robust market.
- Rodríguez-Ballesteros M, Reynoso R, Olarte M, et al. A multicenter study on the prevalence and spectrum of mutations in the otoferlin gene (OTOF) in subjects with nonsyndromic hearing impairment and auditory neuropathy. Hum Mutat. 2008;29(6):823-831.