Lunsotogene Parvec-Cwha

Lunsotogene parvec-cwha is used for one specific type of genetic hearing loss.

• Otarmeni is for children and adults with severe-to-profound or profound sensorineural hearing loss linked to confirmed biallelic OTOF gene variants.
• It is for people with preserved outer hair cell function and no prior cochlear implant in the same ear.
• It is not recommended if imaging shows the inner ear cannot be reached.
• Approval is accelerated and based on improved hearing sensitivity on pure tone audiometry at Week 24.

The OTOF gene makes otoferlin, a protein that hearing cells need to pass sound signals to the hearing nerve. Otarmeni uses a harmless virus vector to deliver a working copy of the gene into the inner ear. It is expressed mainly in inner hair cells, helping them make functional otoferlin and restoring signaling to the auditory nerve, as shown in mouse studies.

Otarmeni is given only as a single-dose infusion into the cochlea (intracochlear), during surgery under general anesthesia. Each treated ear gets its own dose, and both ears are treated in the same session when applicable.

• Oral corticosteroids start the day of infusion, continue for two weeks, then taper over two weeks.
• Antibiotics are given before the incision.
• Vaccines, including against meningitis, are given before treatment.

Your surgical team handles all of this, so a missed dose is not a concern.

In the clinical study of 24 patients, these were the most common side effects:

Less common reactions included temporary balance disorder and wound dehiscence (a wound that opens). Tell your care team about any infection signs, severe dizziness, or facial changes.

• Procedure-related risks are the main concern: vertigo, tinnitus, CSF leak, facial weakness, taste change, numbness around the ear, and fluid or blood collection at the surgical site.
• Infection risks include meningitis, wound infection, mastoiditis, and labyrinthitis.
• You will be monitored after surgery, and problems are managed as needed.

The label does not list specific drug interactions, but vaccine and steroid timing matters.

• Oral corticosteroids are given to help prevent inflammation and immune reactions.
• Vaccines should be given at least 1 month before the first steroid dose and at least 1 month after the last dose.

Not a complete list. The label covers the interactions the manufacturer studied or reported, and our own interaction database does not cover every medicine either. Bring your full medication list, including vitamins and herbal products, to your pharmacist.

• Confirmed biallelic OTOF variants are required.
• Not recommended if the inner ear cannot be accessed, or if you already have a cochlear implant in that ear.
• Pregnancy: no data. Pregnancy status should be verified, and a negative test is needed for sexually active women who can become pregnant.
• Breastfeeding: no data.
• Children: studied in ages 10 months to 16 years.
• Older adults: not studied in people 65 and older.
• No contraindications are listed.

There are no pharmacodynamics data for Otarmeni.

In 15 patients, vector DNA (the gene therapy material) was found in blood, urine, and saliva on Day 1, then was undetectable by Week 2. In stool it could be found through Week 6 and was gone by Week 12. In animal studies, levels were highest in the cochlea, with much lower levels elsewhere.

Store and transport OTARMENI (package 1) frozen at -80°C (-112°F). stored in the refrigerator (2°C to 8°C ) for up to 24 hours or at room temperature (up to 25°C ) for up to 8 hours . Store at room temperature in original carton separately from OTARMENI until time of use.