Sensorion OTOF-GT gene therapy candidate gets FDA rare pediatric designation
Sensorion, a French biotechnology company, has received the rare pediatric disease designation from the US Food and Drug Administration (FDA) for its OTOF-GT gene therapy candidate for the treatment of otoferlin gene mediated hearing loss.
OTOF-GT gene therapy development program targets the restoration of hearing in individuals suffering from otoferlin deficiency.
Sensorion is planning to file a clinical trial application (CTA) for the OTOF-GT candidate in the first half of next year.
Géraldine Honnet — Sensorion Chief Medical Officer said: “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.
“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.”
In September 2022, the European Medicines Agency (EMA) through its Committee for Orphan Medicinal Products (COMP) adopted a positive opinion for an application for orphan drug designation (ODD) of the OTOF-GT gene therapy candidate for the treatment of otoferlin gene mediated hearing loss.
Sensorion’s clinical-stage portfolio includes a phase 2 gene therapy candidate called SENS-401 (Arazasetron). SENS-401 is heading towards a phase 2 proof of concept clinical study in Cisplatin-Induced Ototoxicity (CIO).
The French biotech company is also partnering with Cochlear Limited to undertake a study of SENS-401 in patients with cochlear implantation.
In January 2022, a phase 2 trial of SENS-401 was completed in sudden sensorineural hearing loss.