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FDA Approves Regeneron's Otarmeni Gene Therapy for Hearing Loss

4/25/2026

Summary

FDA Approves Otarmeni

The FDA approved Otarmeni, Regeneron’s gene therapy for hearing loss.
Otarmeni uses a dual adeno-associated virus (AAV) vector to deliver an OTOF gene to the inner ear.
Trials showed hearing improvement in 11 of 12 children, with three achieving normal sensitivity.
Doctors give a one-time injection into both cochleae.
The FDA set a meeting for June 4 on the voucher program after reporting side effects of middle-ear infection, nausea, dizziness, and pain.

Summary

Regeneron Secures MFN

Regeneron secured a Most-Favored-Nation drug-price deal, lowering Praluent prices and providing Otarmeni free to U.S. patients.
President Donald Trump signed the deal and warned of 100% tariffs on drugs that refuse MFN pricing.
Regeneron pledged a $27 billion investment in U.S. research, development and manufacturing through 2029.
Regeneron bought Decibel Therapeutics in 2023 for $109 million, adding the platform that created Otarmeni, first gene therapy cleared via the National Priority Voucher program.

Summary

Regeneron Earns Voucher

Regeneron earned a pediatric disease review voucher worth over $200 million and saw the FDA approve Otarmeni 61 days after filing, the fastest under the National Priority Voucher pilot.
Regeneron must provide post-marketing data on hearing durability, speech development, and quality-of-life to keep the drug on the market.
Leonard Schleifer, CEO of Regeneron, said U.S. launch will be free to “show who we are,” while overseas patients will pay their fair share.

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FDA Approves Otarmeni

The FDA approved Otarmeni, Regeneron’s gene therapy for hearing loss.
Otarmeni uses a dual adeno-associated virus (AAV) vector to deliver an OTOF gene to the inner ear.
Trials showed hearing improvement in 11 of 12 children, with three achieving normal sensitivity.
Doctors give a one-time injection into both cochleae.
The FDA set a meeting for June 4 on the voucher program after reporting side effects of middle-ear infection, nausea, dizziness, and pain.

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