In a pivotal trial, 42% of participants achieved normal hearing using the gene therapy indicated for pediatric and adult patients with severe-to-profound hearing loss due to biallelic OTOF gene variants. The US Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni (lunsotogene parvec-cwha), a gene therapy for pediatric and adult patients with severe-to-profound and profound sensorineural...
Audiologists should be aware that Otarmeni is now FDA-approved for OTOF-related hearing loss and begin familiarizing themselves with candidate identification (genetic testing referral pathways) and post-treatment audiological monitoring protocols, though prescribing authority rests with physicians.
This is the first FDA-approved gene therapy for a genetic form of hearing loss, representing a paradigm shift away from device-based rehabilitation toward biological restoration for eligible patients.
- 01FDA granted accelerated approval to Otarmeni for biallelic OTOF gene variant-related severe-to-profound hearing loss.