Health and Me

Rachel V Thomas

US FDA Approves 1st-Ever Gene Therapy for Treatment of Genetic Hearing Loss

In a groundbreaking move, the US Food and Drug Administration has approved the first-ever dual adeno-associated virus (AAV) vector-based gene therapy to treat hearing loss.

AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound hearing loss.

Developed by American Biotechnology company Regeneron, Otarmeni has been approved for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency more than 90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene.

To date, no disease-modifying treatments exist for OTOF-related deafness. 

“Today’s approval is a significant milestone in the treatment of genetic hearing loss,” said FDA Commissioner Marty Makary, in a statement.

“Through the national priority voucher pilot program, the agency is accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions—such as novel dual vector gene therapies and combination products requiring coordination across multiple offices and centers—in significantly shortened timeframes,” Markary added. 

Importantly, the company has announced that it will offer the therapy free of cost to qualifying individuals, at least during the initial rollout phase. The company cited its commitment to accessibility and patient impact as key reasons behind the decision. 

Hearing loss affects over 430 million people worldwide, with a significant portion caused by genetic mutations. Genetic mutations cause about half of congenital hearing loss. Variants in the OTOF gene account for 2 per cent to 8 per cent of inherited, non-syndromic cases. 

Until now, treatment options have largely been limited to hearing aids or cochlear implants, which assist hearing but do not address the underlying cause. 

Genetic mutations cause about half of congenital hearing loss. Variants in the OTOF gene account for 2 per cent to 8 per cent of inherited, non-syndromic cases. 

The OTOF gene is responsible for producing otoferlin, a protein essential for transmitting sound signals from the inner ear to the brain. Without it, sound cannot be processed, resulting in profound deafness. 

Otarmeni is for patients with preserved outer hair cell function and no prior cochlear implant in the same ear. 

Otarmeni includes a dual adeno-associated virus serotype 1 (AAV1) vector gene therapy administered as a single dose per ear surgically into the cochlea via a syringe and catheter provided in the Administration Kit and connected to an infusion pump. 

The therapy delivers a functional copy of the OTOF gene to inner hair cells to restore otoferlin production and auditory signaling. 

The FDA noted that the common side effects included middle ear infection, nausea, dizziness, and procedural pain. Providers should monitor for surgical complications. It noted that the therapy is not recommended for patients with anatomy that prevents safe access to the inner ear. 

The FDA approval comes after a landmark study, published in the New England Journal of Medicine, showed the benefits of hearing restoration. In trials, 80% of children aged 10 months to 16 years showed real improvement in just 24 weeks. This is not expected in the natural history of the disease without intervention.

Health Conditions A-Z

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otarmeni

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us food and drug administration

regeneron

Hearing Loss

Genetic Hearing Loss

Gene Therapy

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In a groundbreaking move, the US Food and Drug Administration has approved the first-ever dual adeno-associated virus (AAV) vector-based gene therapy to treat hearing loss.AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound hearing loss. Developed by American Biotechnology company Regeneron, Otarmeni has been approved for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency more than 90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene. To date, no disease-modifying treatments exist for OTOF-related deafness. “Today’s approval is a significant milestone in the treatment of genetic hearing loss,” said FDA Commissioner Marty Makary, in a statement.  “Through the national priority voucher pilot program, the agency is accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions—such as novel dual vector gene therapies and combination products requiring coordination across multiple offices and centers—in significantly shortened timeframes,” Markary added. Importantly, the company has announced that it will offer the therapy free of cost to qualifying individuals, at least during the initial rollout phase. The company cited its commitment to accessibility and patient impact as key reasons behind the decision.  How Otarmeni Works Hearing loss affects over 430 million people worldwide, with a significant portion caused by genetic mutations. Genetic mutations cause about half of congenital hearing loss. Variants in the OTOF gene account for 2 per cent to 8 per cent of inherited, non-syndromic cases. Until now, treatment options have largely been limited to hearing aids or cochlear implants, which assist hearing but do not address the underlying cause. Genetic mutations cause about half of congenital hearing loss. Variants in the OTOF gene account for 2 per cent to 8 per cent of inherited, non-syndromic cases. The OTOF gene is responsible for producing otoferlin, a protein essential for transmitting sound signals from the inner ear to the brain. Without it, sound cannot be processed, resulting in profound deafness. Otarmeni is for patients with preserved outer hair cell function and no prior cochlear implant in the same ear. Otarmeni includes a dual adeno-associated virus serotype 1 (AAV1) vector gene therapy administered as a single dose per ear surgically into the cochlea via a syringe and catheter provided in the Administration Kit and connected to an infusion pump. The therapy delivers a functional copy of the OTOF gene to inner hair cells to restore otoferlin production and auditory signaling. Are There Any Side Effects The FDA noted that the common side effects included middle ear infection, nausea, dizziness, and procedural pain. Providers should monitor for surgical complications. It noted that the therapy is not recommended for patients with anatomy that prevents safe access to the inner ear. The FDA approval comes after a landmark study, published in the New England Journal of Medicine, showed the benefits of hearing restoration. In trials, 80% of children aged 10 months to 16 years showed real improvement in just 24 weeks. This is not expected in the natural history of the disease without intervention.