Otarmeni

Otarmeni (lunsotogene parvec-cwha) is a one-time gene therapy used for severe-to-profound and profound sensorineural hearing loss caused by changes in the OTOF gene in certain pediatric and adult patients.

OTOF gene variants can cause a lack of functional otoferlin, a protein needed for communication between sensory cells in the inner ear and the auditory nerve. Otarmeni works by delivering a working copy of the OTOF gene to help restore otoferlin production and auditory signaling, to improve hearing.

Otarmeni is given as a single-dose intracochlear infusion administered surgically into the cochlea.

In the CHORD trial, 80% of evaluable patients treated with Otarmeni had hearing improvement to a level associated with natural hearing and no typical need for cochlear implantation.

Otarmeni is indicated for patients who have:

• Severe-to-profound or profound sensorineural hearing loss, defined as any frequency greater than 90 dB HL
• Molecularly confirmed biallelic variants in the OTOF gene
• Preserved outer hair cell function
• No prior cochlear implant in the same ear

Otarmeni was approved under the FDA’s accelerated approval pathway based on improvement in hearing sensitivity measured by average pure tone audiometry at Week 24. Continued approval may depend on verification and description of clinical benefit in a confirmatory clinical trial.

Patients should be monitored for procedure-related adverse reactions after Otarmeni administration.

Before receiving Otarmeni, patients should discuss the following with their healthcare provider and surgeon:

• Age-appropriate vaccinations, recommended at least 1 month before the first corticosteroid dose and at least 1 month after the last corticosteroid dose
• Vaccination status against meningitis, because meningitis is a known risk of inner ear surgery
• Pregnancy status, if sexually active and able to become pregnant

Serious side effects may occur with the surgery required to administer Otarmeni, including vertigo, ringing in the ears, cerebrospinal fluid leak, partial facial paralysis or weakness, change in taste, meningitis, wound infection, mastoiditis, numbness around the ear, blood or fluid collection at the surgical site, and inflammation of the inner ear.

Before receiving Otarmeni, patients should talk with their healthcare provider and surgeon about vaccination status, including meningitis vaccination, because meningitis is a known risk of inner ear surgery. Age-appropriate vaccinations are recommended at least 1 month before the first corticosteroid dose and at least 1 month after the last corticosteroid dose. Pregnancy status should also be verified in patients who are sexually active and able to become pregnant.

Otarmeni has no listed contraindications.

However, Otarmeni is not recommended for patients whose preoperative imaging shows that access to the inner ear is not feasible. This includes patients with abnormal mastoid pneumatization or clinically significant anatomic variations of the middle ear or inner ear.

The most common adverse reactions reported with Otarmeni, occurring in at least 5% of patients, include:

• Otitis media, also described as middle ear infection
• Vomiting
• Nausea
• Dizziness
• Procedural pain
• Gait disturbance or walking disturbance
• Nystagmus, or rapid involuntary eye movements

Because small amounts of Otarmeni may be present in bodily fluids or waste, materials that may be contaminated should be placed in a sealable bag and disposed of in regular trash for the first 2 weeks after administration. Proper hand hygiene should be used when coming into direct contact with bodily fluids or waste.

Clinical trial results

FDA approval was based on positive results from the pivotal CHORD trial (Study DB-OTO-001; NCT05788536).

In the trial, 24 participants aged 10 months to 16 years (median age 2 years) received a single dose of Otarmeni by intracochlear infusion. Ten participants received treatment in one ear, and 10 received treatment in both ears. 20 had completed efficacy assessments at 24 weeks.

Key results included:

• 80%, or 16 of 20 participants, experienced hearing improvements by pure tone audiometry at a threshold of 70 dB HL or less at 24 weeks.
• One additional participant achieved this threshold by Week 48.
• 70%, or 14 of 20 participants, demonstrated an auditory brainstem response at 90 decibels or less at 24 weeks.
• Among participants followed for 48 weeks, all prior responders maintained a response to therapy.
• 42%, or 5 of 12 participants, followed to 48 weeks, achieved normal hearing that included whispers, defined as 25 dB HL or less.

The CHORD trial is an ongoing Phase 1/2 multicenter, open-label trial evaluating the safety, tolerability, and efficacy of Otarmeni in infants, children, and adolescents with OTOF-related hearing loss.

Yes. Otarmeni received FDA accelerated approval on April 23, 2026, for eligible pediatric and adult patients with severe-to-profound and profound OTOF-related sensorineural hearing loss.

It is the first gene therapy approved for genetic hearing loss and the first FDA-approved example of a gene therapy to restore a neurosensory function to normal levels.

According to the supplied information, Regeneron will provide Otarmeni at no cost to clinically eligible individuals in the United States.

This may not include out-of-pocket costs related to the administration of the therapy. Patients should consult their healthcare provider and/or insurance provider. For access information, patients may contact Regeneron’s OnPath with OTARMENI patient support program at 1-866-500-GENE.

Otarmeni is given as a single-dose intracochlear infusion. It is administered surgically into the cochlea.

The recommended dose for each ear is:

7.2 x 10¹² vector genomes (vg) in a total volume of 0.24 mL, administered by single-dose intracochlear infusion.

Each single-dose vial contains a nominal titer of 3.0 x 10¹³ vg/mL and has an extractable volume of 0.63 mL.

Otarmeni should be administered by a surgeon experienced in intracochlear surgery and trained in the Otarmeni administration process. It should only be administered using the provided Administration Kit for use with Otarmeni.

Oral corticosteroids may be used to help prevent inflammatory and immune responses, and antibiotics may be used to help prevent infection after surgery.