FDA Approves World's First Gene Therapy for Hereditary Hearing Loss After Record 61-Day Review

The U.S. Food and Drug Administration (FDA) has approved the world's first gene therapy for hereditary hearing loss. The developer, Regeneron, has joined the Most Favored Nation (MFN) drug price reduction agreement under the Trump Administration and has decided to provide the therapy free of charge to U.S. patients.

The U.S. Food and Drug Administration (FDA) has approved Regeneron's 'Otarneni,' the world's first gene therapy for treating hereditary hearing loss. Image of a child suffering from hearing loss. Getty Images Korea

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On April 23 (local time), the FDA announced the approval of Otharmeni, developed by Regeneron Pharmaceuticals. The therapy is indicated for pediatric and adult patients with severe to profound sensorineural hearing loss (greater than 90 dB HL at all frequencies) caused by biallelic mutations in the OTOF gene.

This approval was granted just 61 days after the submission of the Biologics License Application (BLA). It marks the sixth approval under the FDA Commissioner's National Priority Review Voucher (CNPV) pilot program and is the first gene therapy to be authorized under this initiative. The approval ties the record for the fastest BLA approval in modern FDA history.

The clinical evidence was based on a single-arm clinical trial involving 24 pediatric patients aged 10 months to 16 years. Among the 20 patients available for efficacy evaluation, 80% (16 patients) demonstrated improvement in hearing. Hearing loss related to the OTOF gene is a disease in which spontaneous recovery of hearing is not expected. The FDA initiated an expedited review immediately after related research results were published in the international journal The New England Journal of Medicine (NEJM).

Otharmeni delivers a functional OTOF gene to inner ear hair cells via an AAV1 vector, thereby restoring production of the otoferlin protein and the transmission of auditory signals. The therapy is administered as a single surgical injection into the cochlea using a syringe and catheter included in the dosing kit. Administration is only possible for patients whose outer hair cell function is preserved and who have no history of cochlear implantation in the same ear.

About half of cases of congenital hearing loss are caused by genetic mutations. Among these, OTOF gene mutations account for 2-8% of hereditary non-syndromic hearing loss. Patients who possess two nonfunctional copies of the gene are unable to produce otoferlin, resulting in a complete block of sound signal transmission. If diagnosis is delayed, the optimal timing for treatment may be missed and language development could be permanently impaired. Prior to the approval of Otharmeni, there were no therapies available to halt the progression of OTOF-related hearing loss.

On the same day, the White House announced that U.S. President Donald Trump had signed an MFN drug price reduction agreement with Regeneron. Accordingly, Regeneron will lower the price of its cholesterol treatment Praluent and apply the same pricing policy to future new drugs.

Regeneron also announced plans to invest $27 billion (about 40 trillion won) in research, development, and manufacturing in the United States through 2029. To date, a total of 17 pharmaceutical companies-including Pfizer, Novartis, and Sanofi-have participated in this policy, collectively accounting for approximately 86% of the branded pharmaceuticals market.

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