FDA approves Regeneron’s Otarmeni gene therapy for rare inherited hearing loss

The FDA has approved Otarmeni, a gene therapy from Regeneron, marking the first therapy cleared under the agency’s national priority voucher program and will be provided to eligible patients at no charge.

Otarmeni is intended for children and adults with severe-to-profound hearing loss caused by mutations in the OTOF gene, which prevents inner-ear hair cells from producing a protein needed to transmit sound signals.

In the pivotal clinical study, participants demonstrated notable improvements, including responding to voices and music, according to trial investigator Eliot Shearer.

The treatment is delivered through a surgical procedure that resembles the implantation of cochlear devices.