Sensorion receives US FDA Rare Pediatric Disease Designation for gene therapy

By Rachel Arthur

- Last updated on GMT


Related tags Sensorion Hearing loss Gene therapy

Sensorion, a biotech specializing in therapies for hearing loss disorders, has been granted Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA) for its lead gene therapy candidate, OTOF-GT.

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.

Sensorion expects to file a Clinical Trial Application (CTA) for OTOF-GT in the first half of 2023. In Europe, it received Orphan Drug Designation last month.

Advancing gene therapies

The French biotech’s portfolio includes small molecule and gene therapy candidates, led by SENS-401 (Arazastron) which is in Phase 2 trials.

It also has a broad strategic collaboration with Institut Pasteur focused on the genetics of hearing. Two gene therapy programs are aimed at correcting hereditary monogenic forms of deafness including OTOF-GT, targeting deafness caused by a mutation of the gene encoding for otoferlin, and hearing loss related to mutation in GJB2​ gene to potentially address important hearing loss segments in adults and children (GJB2-GT).

With OTOF-GT, the large otoferlin protein is delivered to the inner ear using two standard gene therapy vectors (AAV) and assembled in place.

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.

“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.

“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.

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