FDA grants Sensorion Orphan Drug Designation for hearing loss drug

hearing loss sensorion ear cell

Sensorion a clinical-stage biotechnology company has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to OTOF-GT, its lead gene therapy program for hearing loss.

The drug is intended for the treatment of otoferlin gene mediated hearing loss. Sensorion is on track to file a clinical trial application (CTA) 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 prelingual non syndromic hearing loss. During its research, the company found 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 Europe.

Significant regulatory feedback

In September this year, (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 this year on October 11 and on November 7, Sensorion was granted rare pediatric disease designation to OTOF-GT by the US FDA.

Géraldine Honnet, Chief Medical Officer of Sensorion, said: “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.

Sensorion highly focused on life-changing therapies

“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 under this designation 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.

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