FDA approves first gene therapy for inherited hearing loss
The US Food and Drug Administration has approved the first gene therapy for inherited hearing loss, a one-time treatment that showed significant benefits for children born without hearing during a clinical trial, reports BritPanorama.
The therapy targets a rare condition stemming from mutations in the OTOF gene, affecting approximately 50 infants annually in the US. In a clinical trial involving 20 children, 16 exhibited improvements in hearing approximately five months post-treatment, with five of twelve participants monitored for over 11 months achieving near-normal hearing levels.
“It’s miraculous,” stated Kerri, the mother of a trial participant, who wished to remain anonymous. Her 2-year-old son, Miles, received the therapy in May. “You go from being told your child’s profoundly deaf and may only ever hear with technology to your child’s hearing right alongside his friends. … This is just amazing.”
Developed by Regeneron, the gene therapy will be provided free of charge to patients in the US. The company, which previously announced plans regarding the therapy’s pricing, highlighted its intent to demonstrate how biotechnology can profoundly benefit patients, as explained by Dr. George Yancopoulos, the co-founder and president of Regeneron.
The treatment was expedited under a new National Priority Voucher program aimed at accelerating the review process for promising therapies. Regeneron is also expected to participate in an announcement at the White House concerning drug pricing agreements designed to align US drug costs with those in Europe and Asia.
While Regeneron has indicated plans to seek regulatory approval in other nations, it did not clarify whether the therapy would also be provided at no cost to patients outside the US. The treatment, named Otarmeni, involves a surgical procedure akin to cochlear implantation and has demonstrated safety in clinical trials, with side effects typical of surgical interventions. However, the overall expenses may still encompass out-of-pocket costs related to the surgical procedure, which Regeneron does not perform.
This development marks a significant advancement in the treatment of inherited hearing loss, reflecting ongoing innovation in genetic therapies.
