FDA Approves World's First Gene Therapy for Hearing Loss

According to the WSJ on the 23rd, the FDA has approved the first gene therapy to restore hearing. The New York Times reported that this new gene therapy can restore hearing in children with rare hearing loss. Fierce Pharma evaluated that Regeneron ushered in a new era with the approval of the first gene therapy, and Reuters also reported that Regeneron secured FDA approval for the first gene therapy for hereditary hearing loss. The journal Nature stated that a multicenter gene therapy for OTOF-related hearing loss was followed up for up to 2.5 years.

FDA's Ultra-Fast Approval and the Emergence of 'Otarmeni'

The U.S. Food and Drug Administration (FDA) announced through an official statement on the 23rd local time that it has granted final approval to 'Otarmeni (ingredient name: lunsotogene parvec-cwha)', developed by Regeneron Pharmaceuticals. This drug is the first-ever dual adeno-associated virus (AAV) vector-based gene therapy for pediatric and adult patients suffering from severe sensorineural hearing loss due to biallelic mutations in the OTOF gene.

This approval was achieved in just 61 days after the submission of the Biologics License Application (BLA) through the FDA's 'Commissioner's National Priority Voucher (CNPV)' pilot program. This accomplishment ties the record for the fastest BLA approval in modern FDA history, making it the first gene therapy approved under this program. FDA Commissioner Marty Makary emphasized, "This approval is a significant milestone in the treatment of hereditary hearing loss," and added, "It proves that the FDA can drastically shorten the review period even for the most complex new drug submissions."

Groundbreaking Mechanism of Action and Overwhelming Clinical Results

Approximately half of congenital hearing loss cases are caused by genetic mutations, and among them, OTOF gene mutations lead to a deficiency in the 'otoferlin' protein, which is essential for transmitting sound signals to the brain. Otarmeni works by being injected once into the cochlea to deliver a normal copy of the OTOF gene to the inner hair cells, thereby normalizing the production of the otoferlin protein and restoring auditory signal transmission.

The clinical trial that served as the key basis for the FDA approval was conducted on 24 pediatric patients aged between 10 months and 16 years. Out of the 20 patients evaluated for efficacy, a staggering 80% showed significant hearing improvement, and 42% of them demonstrated the remarkable result of recovering to normal hearing levels capable of hearing even whispers. According to Regeneron, this hearing restoration effect has been sustained for at least two years so far.

Declaration of Completely 'Free' Provision for U.S. Patients

Another element that caught public attention in this announcement is the unconventional pricing policy. Regeneron declared that it will provide this groundbreaking gene therapy completely free of charge to patients in the U.S. OTOF gene mutation-related hearing loss is an ultra-rare disease that occurs in only about 50 newborns in the U.S. each year. Considering that single-dose gene therapies for rare diseases are typically priced at astronomical amounts reaching millions of dollars, this is an unprecedented decision. George Yancopoulos, co-founder of Regeneron, explained the intent, stating, "We wanted to set an example of how biotechnology can deliver the gift of 'hearing' to people."

However, while the therapy itself is free, it involves a surgical procedure to access the cochlea for drug injection, so patients may have to bear the costs associated with the hospital surgery and procedure. Major side effects reported include otitis media, nausea, dizziness, and pain at the procedure site, but the clinical trials have proven it to be generally safe.