Lunsotogene Parvec: First Approval.

Lunsotogene parvec (lunsotogene parvec-cwha; OTARMENI™) is a dual adeno-associated virus (AAV) gene transfer therapy developed by Regeneron Pharmaceuticals for the treatment of profound, congenital hearing loss caused by OTOF variants. Delivery of the OTOF transgene to the inner hair cells facilitates production of functional otoferlin protein to restore synaptic transmission to the auditory nerve. Lunsotogene parvec received its first approval on 23 April 2026 in the USA for the treatment of paediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency > 90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function and no prior cochlear implant in the same ear. This article summarises the milestones in the development of lunsotogene parvec leading to this first approval for OTOF-associated sensorineural hearing loss.