FDA Grants Rare Pediatric Disease Designation to Sensorion’s Gene Therapy for Otoferlin Gene-Mediated Hearing Loss

Rare Daily Staff

The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to French biotech Sensorion’s lead gene therapy candidate, OTOF-GT, 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 AAV gene therapy vectors and assembled in place. Patients with OTOF mutations suffer from severe to profound sensorineural prelingual non syndromic hearing loss. There are currently no approved therapies for otoferlin deficiency, which could be responsible for up to 8 percent of all cases of congenital hearing loss, with 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 in the first half of 2023. OTOF-GT was granted Orphan Drug designation by the European Medicines Agency in October 2022.

“This important regulatory designation will support us in advancing this potentially transformative therapy to patients,” said Géraldine Honnet, chief medical officer at Sensorion. “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 United States. 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. Most recently, Marinus Pharmaceuticals sold its voucher for $110 million to Novo Nordisk.

Photo: Géraldine Honnet, chief medical officer at Sensorion