Sensorion granted rare pediatric disease designation by FDA for lead therapy gene

November 7, 2022 - 2 minutes
Picture/Sensorion

Sensorion has announced that the US Food and Drug Administration (FDA) has granted rare pediatric disease designation to its lead therapy gene candidate, OTOF-GT.

The gene therapy is intended for the treatment of otoferlin gene mediated hearing loss.

Sensorion’s OTOF-GT gene therapy development program aims to restore hearing in people living with otoferlin deficiency, one of the most common forms of congenital deafness. The otoferlin protein is large and is delivered to the inner ear using two standard gene therapy vectors (AAV) and assembled in place.

About Sensorion’s lead therapy gene candidate

Patients with OTOF mutations suffer from severe to profound sensorineural prelingual non syndromic hearing loss. Otoferlin deficiency could be responsible for up to 8% of all cases of congenital hearing loss, around 20,000 people are affected in the United States and in Europe.

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.

“We are really pleased that the FDA has acknowledged the urgent need to develop solutions for this condition, for which there are currently no approved therapies. This important regulatory designation will support us in advancing this potentially transformative therapy to patients,” said Géraldine Honnet, chief medical officer of Sensorion.

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“This is a key milestone for our important gene therapy franchise, which offers the potential for permanent solutions for these debilitating conditions and is increasingly central to Sensorion’s strategic roadmap. The designation is another key piece in the puzzle that helps advance Sensorion towards clinical development in OTOF-GT in 2023.”  

The FDA grants rare pediatric disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the USA.

Under this 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.

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