(CercleFinance.com) – Sensorion said on Wednesday that its gene therapy for the treatment of hearing loss linked to the otoferlin gene had obtained orphan drug designation from the US health authorities.
The ‘OTOF-GT’ program developed by the company aims to restore hearing to patients with otoferlin deficiency, a feature that may be responsible for up to 8% of all cases of congenital hearing loss.
About 20,000 people are affected in the United States and Europe.
This orphan drug designation by the Food & Drug Administration (FDA) should allow the biotechnology company to benefit from an accelerated regulatory process.
Sensorion says it is on track to file a clinical trial authorization application for OTOF-GT in the first half of 2023.
In September, the company had already received the favorable opinion of the European Medicines Agency (EMA) for an application for orphan drug designation.
Following this announcement, the action of the ‘biotech’ progressed by 4.6% on Wednesday at the Paris Stock Exchange.