Sensorion Announces US FDA Grants Rare Pediatric Disease Designation to OTOF-GT for the Treatment of Otoferlin Gene-Mediated Hearing Loss
Sensorions 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.
- Sensorions 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.
- Patients with OTOF mutations suffer from severe to profound sensorineural prelingual non syndromic hearing loss.
- 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.