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

Otarmeni (DB-OTO)

Restore hearing in OTOF-deaf patients. Deliver functional OTOF gene to inner ear hair cells, enabling sound perception without implants.

DeployStage 4 of 5

FDA accelerated approval April 23, 2026. Clinical use begins; long-term follow-up ongoing.

Updated Sep 2026Checked 11 Oct0 updates this week

Milestones

Next · Commercial availability in U.S.

CHORD clinical trial completed2025Complete.
FDA accelerated approval23 Apr 2026Complete.
Commercial availability in U.S.Target 23 Apr 2026Current milestone.
Long-term safety follow-upTarget 2027Not yet reached.

Most important updates

Upcoming

23 Apr 2026Commercial availability in U.S. (next)
2027Long-term safety follow-up
2027International regulatory approvals
2027Expansion to other OTOF patient populations

Current obstacles

Limited to biallelic OTOF mutationsOnly patients with two mutated OTOF gene copies are candidates. OTOF mutations <5% of genetic hearing loss, limiting addressable population.
Procedural risksGeneral anesthesia and cochlear surgery carry rare but serious risks: infection and hearing loss in treated ear.

Physics limits

One-way therapy in post-mitotic cellsInner ear hair cells do not regenerate. Once treated, the gene remains; if hearing again deteriorates from other causes (aging, noise), additional therapy will not help.
AAV immune responsesSome patients develop antibodies to the AAV vector, potentially limiting re-dosing or treatment of the other ear. Frequency and clinical impact are still being studied.

How it works

3 parts

Vector

AAV carries the OTOF gene

Engineered adeno-associated virus (AAV) delivers OTOF DNA to inner ear cells. AAV is small and non-integrating, minimizing off-target effects.

Delivery

Cochlear infusion under anesthesia

Procedure similar to cochlear implant surgery. One treatment; no repeated doses in trials.

Effect

Hair cells produce otoferlin protein

Inner ear hair cells translate OTOF into otoferlin, a protein essential for sound transmission between cell compartments.

Spec sheet

Spec	Otarmeni (DB-OTO)
Patient population	Severe-to-profound OTOF-related hearing loss^R (reported)
CHORD trial participants	25 (ages 10 months to 16 years)^R (reported)
Improved hearing at 24 weeks	80% (20 of 25 evaluable)^R (reported)
Participants achieving normal hearing	42% (at extended follow-up)^R (reported)

^R reported by the company

Update log

2 updates

Sep 2026

Minor: Regeneron presents Otarmeni long-term safety and efficacy data at medical conferencesPaperResearch

Thu 23 Apr

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

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 trials, 80% showed improved hearing; 42% achieved normal hearing levels.

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

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

Spec

Otarmeni (DB-OTO)

Patient population

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

CHORD trial participants

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

Improved hearing at 24 weeks

80% (20 of 25 evaluable)^R (reported)

Participants achieving normal hearing

42% (at extended follow-up)^R (reported)