The FDA has granted accelerated approval to Regeneron Pharmaceuticals’ Otarmeni
(lunsotogene parvec-cwha) as the first gene therapy designed to restore a neurosensory function to normal levels.
Otarmeni is an adeno-associated virus vector-based gene therapy indicated for treating children and adults with severe-to-profound and profound sensorineural hearing loss, defined as any frequency >90 decibel hearing level [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.
Otarmeni (formerly DB-OTO) is the first and only in vivo gene therapy indicated for OTOF-related hearing loss. Regeneron said it will make Otarmeni available for free in the U.S.
The FDA based its accelerated approval decision on the improvement of hearing sensitivity as measured by average pure tone audiometry (PTA) at week 24 during the Phase I/II CHORD trial (NCT05788536). Twenty participants ages 10 months to 16 years…
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