Sensorion, a pioneering clinical-stage biotechnology company dedicated to developing innovative therapies to restore, treat and prevent hearing loss, announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the therapy company’s most advanced gene therapy, OTOF-GT, a gene therapy for the treatment of hearing loss related to the otoferlin gene. Sensorion is on track to apply for clinical trial authorization for OTOF-GT in the first half of 2023.
Sensorion’s dual AAV vector gene therapy product development program, OTOF-GT, aims to restore hearing in patients with otoferlin deficiency. Patients with mutations related to otoferlin deficiency have severe to profound nonsyndromic prelingual sensorineural hearing loss. Otoferlin deficiency may be responsible for nearly 8% of all cases of congenital hearing loss and approximately 20,000 people are affected in the United States and Europe1.
Sensorion continues to advance its preclinical and clinical development plans for OTOF-GT and is on track to submit for clinical trial authorization in the first half of 2023. In September 2022, the Company received a favorable opinion from the European Agency for medicines (EMA) for an application for orphan drug designation (ODD). The decision was adopted by the European Commission on October 11, 2022. Sensorion also obtained Pediatric Rare Disease designation for OTOF-GT from the US FDA on November 7, 2022.
“We are very pleased to receive this important regulatory notice from the FDA, following the recent receipt of pediatric rare disease designation for OTOF-GT. The orphan drug designation will support the advancement of this gene therapy development program which has the potential to help patients with a disease for which there is currently no approved cure,” said Géraldine Honnet, director of Sensorion Medical.” We are very pleased to have achieved this milestone and remain highly focused on developing the most promising candidates to develop therapies that have the potential to change patients’ lives by restoring, treating and preventing hearing loss disorders.”
The FDA grants orphan drug designation to encourage the development of therapies to treat, diagnose, or prevent so-called rare diseases that affect fewer than 200,000 people in the United States. This status allows Sensorion to benefit from numerous advantages, including in particular a seven-year marketing exclusivity period following marketing authorization, development consultancy, tax credits for expenses related to trials clinics and waivers from some FDA fees.
Under the “rare pediatric disease” designation, a sponsor who receives approval for a drug or biological product for a “rare pediatric disease” is eligible for a voucher that can be redeemed for expedited subsequent application review of marketing for a different product or sold to another sponsor for an accelerated review of their marketing application, which represents an opportunity for which there is a dynamic market.
Source and picture: Sensation
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