USANew treatments and approvals
Lunsotogene parvec approved in the USA as the first gene therapy for congenital deafness caused by the OTOF gene
A decision by a medicines regulator (FDA, European Commission, etc.). The regulator has reviewed the clinical studies behind the drug. For accelerated or conditional approvals, the lasting benefit is still being confirmed.
The US Food and Drug Administration approved the first gene therapy for severe hearing loss caused by the OTOF gene. The drug is given once, surgically, into the inner ear. The approval is based on a single study that included 24 children aged 10 months to 16 years. Hearing improved in 80% of the 20 children whose response was evaluated. This is an accelerated approval; how long the effect lasts will be monitored.
What does it mean for patients and families?
This treatment is only for people who have changes in both copies of OTOF and whose inner ear hair cells are working. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes. Eligibility is decided with an ear, nose and throat doctor and a genetics doctor.
Source
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: OTOF-related congenital hearing loss
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.