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The U.S. Food and Drug Administration approved Otarmeni, a gene therapy developed by Regeneron Pharmaceuticals, for patients with severe-to-profound hearing loss linked to variants in the OTOF gene. It is the first disease-modifying treatment for OTOF-related deafness.

The U.S. Food and Drug Administration approved Otarmeni, a gene therapy developed by Regeneron Pharmaceuticals, for patients with severe-to-profound hearing loss linked to variants in the OTOF gene. It is the first disease-modifying treatment for OTOF-related deafness.

The FDA approved Otarmeni 61 days after Regeneron filed for a biologics license, making it the sixth approval under the Commissioner's National Priority Voucher pilot program and the first gene therapy approved through the initiative.

The FDA approved Otarmeni 61 days after Regeneron filed for a biologics license, making it the sixth approval under the Commissioner's National Priority Voucher pilot program and the first gene therapy approved through the initiative.

The approval was based on a clinical trial of 24 pediatric patients aged 10 months to 16 years. Of the 20 evaluable for efficacy, 80% experienced hearing improvement, with five of 12 followed for at least 11 months achieving essentially normal hearing.

The approval was based on a clinical trial of 24 pediatric patients aged 10 months to 16 years. Of the 20 evaluable for efficacy, 80% experienced hearing improvement, with five of 12 followed for at least 11 months achieving essentially normal hearing.

Otarmeni is administered as a one-time surgical procedure, delivering a functional copy of the OTOF gene into the cochlea via a syringe and catheter. Common side effects included middle ear infection, nausea, dizziness and procedural pain.

Otarmeni is administered as a one-time surgical procedure, delivering a functional copy of the OTOF gene into the cochlea via a syringe and catheter. Common side effects included middle ear infection, nausea, dizziness and procedural pain.

Regeneron announced it will provide Otarmeni at no cost to patients in the United States, though patients may be responsible for fees related to the surgical procedure. Gene therapies for rare diseases have previously carried price tags of up to $2 million to $3 million per patient.

Regeneron announced it will provide Otarmeni at no cost to patients in the United States, though patients may be responsible for fees related to the surgical procedure. Gene therapies for rare diseases have previously carried price tags of up to $2 million to $3 million per patient.

OTOF gene variants account for 2% to 8% of inherited, non-syndromic hearing loss cases, affecting an estimated 20 to 50 newborns in the U.S. each year. Researchers say the approval could spur development of gene therapies for more than 150 known genetic causes of hearing loss.

OTOF gene variants account for 2% to 8% of inherited, non-syndromic hearing loss cases, affecting an estimated 20 to 50 newborns in the U.S. each year. Researchers say the approval could spur development of gene therapies for more than 150 known genetic causes of hearing loss.

FDA Approves First Gene Therapy for Genetic Deafness

FDA Approves First-Ever Gene Therapy tofor RestoreGenetic HearingDeafness

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FDA Approves First Gene Therapy for Genetic Deafness

FDA Approves First-Ever Gene Therapy tofor RestoreGenetic HearingDeafness

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