Regeneron Announces FDA Approval of Otarmeni, the World’s First Gene Therapy for Genetic Hearing Loss

(Source: Yaoyan Network)

April 23,Regeneron announced,United StatesFDA ApprovedIts Gene Therapy Otarmeni Under Research Receives Regulatory Approval for Market Launch，This is a gene therapy for rare hereditary deafness, treatmentChildren and adult patients with severe to profound or profound sensorineural hearing loss associated with biallelic mutations in the OTOF gene。

This approval was granted under the FDA's new Priority Voucher Program, markingThe First Gene Therapy for Hereditary Hearing Loss Enters the Market.

The approval was based on the pivotal CHORD clinical trial (Phase I/II, multicenter, open-label study), which enrolled a total of 20 subjects aged between 10 months and 16 years.

Efficacy Results at 24 Weeks

Natural Hearing Recovery: 80% of subjects achieved a pure-tone audiometric threshold of ≤70dB HL, meeting the clinically defined standard for natural hearing without reliance on cochlear implants.

Objective Restoration of Hearing Function: 70% of subjects showed Auditory Brainstem Response (ABR) thresholds ≤90dB, objectively confirming effective restoration of hearing function.

48-Week Long-Term Follow-Up Results

All treatment responders maintained their efficacy continuously. Among them, 42% of participants fully recovered their hearing (pure-tone audiometry threshold ≤25dB HL) and could clearly recognize whispering sounds.

Regeneron stated that the Otarmeni therapy will be freely accessible to patients in the United States. The company also plans to announce a drug pricing agreement with the White House this afternoon, which will lower the prices of drugs under the U.S. Medicaid program based on drug prices in other developed countries, and align future drug prices in the U.S. with those in these developed nations.

In this indication area, global competition is becoming increasingly intense. Companies in ChinaShanghai Dinxin Gene Technology and Eli LillyCollaboration is underway to research therapies for this disease (inferred from the context as hereditary deafness). In early clinical trials, the therapy has shown potential to restore hearing in children.