Some types of hearing loss are caused by inherited gene changes that affect hearing from birth. People with these conditions may have severe hearing loss very early in life, and they often rely on hearing support devices, such as cochlear implants, to help hear sounds and develop speech and language skills.
In April 2026, the FDA approved Otarmeni (lunsotogene parvec-cwha), the first gene therapy approved for certain people with severe or complete hearing loss caused by specific gene changes.
Continue reading to learn how Otarmeni works, what researchers found in clinical studies, and what to expect with treatment.
Otarmeni Side Effects and Interactions: What to Expect and How to Manage Them
Is Otarmeni Right for Me?
Otarmeni is a treatment for children and adults with very severe hearing loss or complete hearing loss. This type of hearing loss is called sensorineural hearing loss, which means it is caused by problems in the inner ear or the hearing nerve.
According to its FDA-approved use, you must meet a few specific conditions to be able to receive Otarmeni:
- Your hearing loss is very severe (greater than 90 decibels), which means you cannot hear most sounds, even very loud ones.
- Your hearing loss is caused by changes in a specific gene called the OTOF gene, which must be confirmed with genetic testing.
- Some parts of your inner ear (called outer hair cells) are still working.
- You have not already received a cochlear implant in the ear being treated.
Before Otarmeni treatment, you may need to:
- Get vaccines recommended for your age
- Receive vaccination against germs that can cause meningitis
- Start corticosteroid treatment to help lower your risk of inflammation
- Receive antibiotics before surgery to help prevent infection
Otarmeni is a one-time treatment, delivered directly into the inner ear through a surgical procedure. This means the medicine is placed inside the cochlea (intracochlear infusion), which is the part of the ear responsible for hearing.
The procedure must be done by a surgeon who is specially trained and experienced in this type of ear surgery. If both ears need treatment, the surgeon may treat both ears during the same surgery session.
Otarmeni is not recommended for people whose inner ear anatomy could make the procedure unsafe. A health care provider can help decide if Otarmeni is a good option for you or your child.
How Does Otarmeni Work for Hearing Loss?
Otarmeni is a type of gene therapy, which means it works by delivering a healthy version of a gene into the body to help fix an underlying problem.
In this case, Otarmeni is designed to treat hearing loss caused by changes in the OTOF gene. This gene normally helps the body make a protein called otoferlin, which plays an important role in hearing. Otoferlin helps sound signals move from special cells in the inner ear to the hearing (auditory) nerve so the brain can process sound. When the OTOF gene does not work properly, the body cannot make enough otoferlin. As a result, sound signals cannot travel to the brain the way they should.
Otarmeni uses a harmless virus (called an adeno-associated virus) as a delivery system. This virus has been modified so it cannot cause disease. Instead, it carries a working copy of the OTOF gene into specific cells in the inner ear.
The gene is delivered mainly to the inner hair cells in the ear, which play a key role in hearing. Once inside these cells, the gene helps the body make the otoferlin protein. This may help restore the ability of the ear to send sound signals to the hearing nerve.
How Was Otarmeni Studied for Hearing Loss?
Otarmeni was studied in an ongoing clinical trial called CHORD. The study included children with severe to complete (profound) hearing loss caused by inherited OTOF gene changes.
The study included 12 children:
- Their ages ranged from about 10 months to 16 years.
- Three children received Otarmeni treatment in both ears during the same surgery.
- Nine children received Otarmeni treatment in one ear, and some children also had a cochlear implant in the ear that was not treated with Otarmeni.
In this study, researchers measured hearing improvement using several tests. The main test looked at hearing sensitivity using pure-tone audiometry (PTA). Lower scores on this test mean better hearing. Researchers also measured auditory brain-stem responses, which look at how sound signals travel from the ear to the brain.
The main goal of the study was to see how many children reached hearing levels that could allow natural hearing without needing a cochlear implant. Researchers also looked at speech perception and language development in some children.
What Benefits Were Seen With Otarmeni?
Many children in the study had improved hearing after receiving Otarmeni. Researchers measured hearing changes using PTA, which tests a person’s ability to hear soft sounds. (Lower PTA scores mean better hearing.) Before treatment, all children had profound hearing loss, with PTA levels above 90 decibel hearing level (dB HL).
At 24 weeks after treatment, 9 of the 12 children (75%) reached PTA levels of 70 dB HL or lower, which was the study’s main goal. This level of hearing is usually good enough to avoid needing a cochlear implant.
Of these nine children, six could hear soft speech without assistive devices, and three reached hearing levels considered normal. Researchers did not observe similar hearing improvements in ears that were not treated.
The study also found that 9 of the 12 children developed improved auditory brain-stem responses by 24 weeks. This means that sound signals were traveling through the hearing nerve pathway more effectively.
Keep in mind that your results may differ from what was seen in studies.
Note that the FDA approved Otarmeni through the accelerated approval pathway, and additional studies are ongoing to confirm long-term benefits.
Does Otarmeni Work Long Term?
Researchers are still studying how long the benefits of Otarmeni may last. It is a one-time treatment expected to have long-term benefits based on how the medicine (gene therapy) is designed.
In the participants who were followed for up to 72 weeks, hearing levels generally stayed stable or improved further. Long-term monitoring is still ongoing to better understand the effect and safety of Otarmeni over time.
As with most medicines, Otarmeni may not work the same way for everyone. In the clinical study, most participants (75%) had improved hearing after treatment. However, some children had smaller improvements, and one participant did not have any meaningful hearing improvement and later received a cochlear implant.
Researchers do not yet know why some people have better results than others. Your health care provider will continue monitoring your hearing after Otarmeni treatment.
Is There Any Cost Assistance Available?
The maker of Otarmeni, Regeneron, provides the therapy at no cost to eligible patients in the United States through its OnPath with Otarmeni patient support program. Some patients may still have out-of-pocket costs related to the administration of the treatment. To learn more, visit this website or call 866-500-4363 to speak to a representative about eligibility and support.