Deaf toddler treated with Regeneron’s Otarmeni

A 16-month child with auditory neuropathy spectrum disorder (ANSD) became the first patient at Boston Children’s Hospital to undergo treatment with Regeneron’s gene therapy, Otarmeni.

Otarmeni, which was granted accelerated approval by the FDA in April, is the first and only in vivo gene therapy for OTOF-related hearing loss. Regeneron is offering the therapy for free in the U.S.

Otarmeni is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. The treatment aims to deliver a working copy of the OTOF gene to replace the non-functional otoferlin protein using a modified, non-pathogenic virus that is delivered via an infusion into the cochlea under general anesthesia (similar to the procedure used for cochlear implantation).

The patient’s surgery was successful and he is recovering. While time is needed to see if his full hearing is restored, the child’s parents report they have already seen improvements in his awareness of surroundings.