Otarmeni (DB-OTO) by Regeneron Pharmaceuticals: status and timeline

Otarmeni (DB-OTO)

Give natural hearing, with one treatment, to people born deaf from OTOF mutations, instead of a lifelong cochlear implant. It is the first proof that a gene therapy can switch hearing on in a human ear.

DeployStage 4 of 5

FDA accelerated approval April 2026; free in the US; durability and speech data still owed to the FDA.

Updated 23 Apr 2026Checked 11 Oct0 updates this week

Milestones

Next · US use at no cost to eligible patients

Regeneron agrees to buy Decibel, DB-OTO's developerAug 2023Complete.
CHORD interim data in NEJM: 11 of 12 children improved12 Oct 2025Complete.
Licence application filed under national priority voucherFeb 2026Complete.
FDA accelerated approval as Otarmeni23 Apr 2026Complete.
US use at no cost to eligible patientsTarget Apr 2026Current milestone.
Confirmatory data: durability, speech, quality of lifeNot yet reached.

Most important updates

Upcoming

Apr 2026US use at no cost to eligible patients (next)

Current obstacles

Conditional approvalTo keep the label, Regeneron must show the hearing gains last and lead to speech development and better quality of life.
Small, hard-to-find patient poolAbout 200,000 people worldwide have OTOF deafness, and the treated ear must not already have a cochlear implant.
Surgery on the inner earCommon side effects: middle ear infection, nausea, vomiting, dizziness, procedure pain. Rarer risks include meningitis, spinal fluid leak and facial weakness.

Physics limits

Needs living hair cellsAdding a gene only helps when the hair cells are still alive and lack one protein. Most deafness comes from dead hair cells or other genes, which this kind of therapy cannot rescue.
Language has a timing windowThe brain's language circuits mature in early childhood, so hearing restored later may not bring normal speech. Years of follow-up are needed to see how much age at treatment matters.

How it works

4 parts

Problem

Hair cells that cannot pass the signal on

Inner hair cells turn sound into signals for the hearing nerve. They need the protein otoferlin to pass them on; two broken OTOF genes leave a child deaf.

Vector

The gene split across two viruses

OTOF is too large for one adeno-associated virus (AAV), so it is split across two; a hair cell that takes up both rebuilds the full gene.

Delivery

One infusion into the cochlea

Under general anesthesia, surgeons infuse it into the cochlea, much like cochlear implant surgery. The trial treated one ear first; its Part B treats both.

Effect

Hearing within weeks, still rising

Children began responding to sound within weeks. In 8 followed 36 weeks or more, hearing kept improving or held steady for up to 72 weeks.

Spec sheet

Spec	Otarmeni (DB-OTO)
Patient population	Severe-to-profound hearing loss, biallelic OTOF^R (reported)
CHORD trial participants	25 (ages 10 months to 16 years)^R (reported)
Hearing ≤70 dB HL at 24 weeks	80% (16 of 20)^R (reported)
Brainstem response ≤90 dB, week 24	70% (14 of 20)^R (reported)
Normal hearing (≤25 dB HL), week 48	42% (5 of 12)^R (reported)
Filing to approval	61 days^R (reported)
US price	Free (administration costs extra)^R (reported)

^R reported by the company

Update log

2 updates

Thu 23 Apr

Major: FDA grants accelerated approval to Regeneron's Otarmeni, the first gene therapy for inherited deafnessPressRegulatory

Sun 12 Oct 2025

Major: NEJM: Regeneron's DB-OTO gave clinically meaningful hearing to 11 of 12 children born deaf from OTOF mutationsPressResearch

About Regeneron Pharmaceuticals

The team behind Otarmeni (DB-OTO)

Regeneron Pharmaceuticals

OTOF gene therapy, monoclonal antibodies, Eylea, Dupixent

Regeneron Pharmaceuticals, based in Tarrytown, NY and traded on Nasdaq, received FDA approval for Otarmeni (DB-OTO) in April 2026, the first gene therapy for inherited deafness. It advances programs in gene editing, RNA medicines, and synthetic biology.

Regeneron has $10B+ annual revenue, spanning monoclonal antibodies (Eylea, Dupixent), RNA therapeutics, and gene therapies.
Otarmeni is the first FDA-approved gene therapy for inherited deafness, delivering OTOF gene to inner ear hair cells.
In CHORD, 80% (16 of 20) reached the hearing target at 24 weeks; 5 of 12 had normal hearing at 48 weeks.

regeneron.com (opens regeneron.com)Company profile →

Founded: 198838 yrs
Headquarters: United States
Status: Public
Valuation: public
Works in: BiotechGene therapyNeurotech
Coverage: 1 program · 2 updateslatest 23 Apr 2026checked 11 Oct
Customers: FDA
People: George D. YancopoulosPresident and Chief Scientific Officer

Spec

Otarmeni (DB-OTO)

Patient population

Severe-to-profound hearing loss, biallelic OTOF^R (reported)

CHORD trial participants

25 (ages 10 months to 16 years)^R (reported)

Hearing ≤70 dB HL at 24 weeks

80% (16 of 20)^R (reported)

Brainstem response ≤90 dB, week 24

70% (14 of 20)^R (reported)

Normal hearing (≤25 dB HL), week 48

42% (5 of 12)^R (reported)

Filing to approval

61 days^R (reported)

US price

Free (administration costs extra)^R (reported)