The US FDA has approved lunsotogene parvec, a gene therapy for the treatment of pediatric and adult patients with severe-to-profound sensorineural hearing loss (any frequency >90 dB HL). The indication is limited to individuals with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the affected ear. This adeno-associated virus vector–based therapy is the first and only gene therapy approved by the FDA for genetic hearing loss under the accelerated approval pathway.
Clinical evidence Efficacy is supported by an ongoing single-arm clinical trial involving 24 patients aged 10 months to 16 years. At week 24, 80% of patients achieved a hearing sensitivity threshold of ≤70 dB HL, as assessed by pure tone audiometry (PTA), and 70% demonstrated an auditory brainstem response to a click (broadband sound) stimulus of…
