The European Medicines Agency (EMA) through its Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion for a treatment for otoferlin gene mediated hearing loss.
Clinical-stage biotech, Sensorion, announced the news today (September 12) that its application for an Orphan Drug Designation (ODD) for its lead therapy gene candidate, OTOF-GT will now be passed to the European Commission.
The European Commission will issue a decision within 30 days of receipt of the COMP positive opinion for OTOF-GT which aims to restore hearing in people living with otoferlin deficiency.
Sensorion says patients with mutations in OTOF suffer from severe to profound sensorineural prelingual non syndromic hearing loss. Otoferlin deficiency could be responsible for up to 8 per cent of all cases of congenital hearing loss, around 20,000 people are affected in the US and Europe.
Valérie Salentey, regulatory affairs and quality assurance director of Sensorion, said: “This important regulatory feedback is great news as the ODD will support us in advancing our OTOF-GT development program to bring this innovative therapy to the patients who need it most.
“We have continued to progress with preclinical and clinical development plans for OTOF-GT and are on track to file a Clinical Trial Application (CTA) for the program in H1 2023. This is a key part of our growing gene therapy franchise for the restoration of auditory function in a number of indications through our collaboration with Institut Pasteur.”
ODD is designed to encourage the development of medicines intended for the treatment of life-threatening or chronically debilitating diseases that are rare – defined as affecting fewer than five in 10,000 people in the EU.
The designation provides companies with certain advantages and incentives, including protocol assistance, fee reductions, differentiated evaluation procedures for Health Technology Assessments (HTAs) in certain countries and 10 years’ market exclusivity.
Sensorion has in its clinical-stage portfolio one phase 2 product called SENS-401 (Arazasetron) progressing in a planned phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity (CIO) and, with partner Cochlear Limited, a study of SENS-401 in patients scheduled for cochlear implantation.
The company has entered into a broad strategic collaboration with Institut Pasteur focused on the genetics of hearing.
Sensorion’s gene therapy programs
It has two gene therapy programs 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.
It hopes to potentially address important hearing loss segments in adults and children.
In 2020, Sensorion announced it had been forced to delay its phase 2 trial in people with hearing loss due to slow patient recruitment amid the pandemic.