"Otarmeni, made by Regeneron, is the first gene therapy approved by the FDA for treating autosomal recessive deafness caused by mutations in the OTOF gene."
"The FDA's approval of Otarmeni was fast-tracked under the National Priority Voucher program, which aims to expedite therapies for rare diseases with unmet medical needs."
"Experts believe that Otarmeni's approval represents a significant advancement in the treatment of deafness, with Zheng-Yi Chen calling it 'an historical event, a landmark.'"
Hearing loss affects many globally, with some cases linked to genetic mutations like those in the OTOF gene. Otarmeni, developed by Regeneron, is the first FDA-approved gene therapy for this condition. Its approval was expedited through the National Priority Voucher program, aimed at rare diseases. Approximately 50 newborns in the U.S. are affected annually by this condition. Regeneron plans to provide Otarmeni for free to eligible individuals, marking a significant advancement in deafness treatment, as noted by experts in the field.
Read at InsideHook
Unable to calculate read time
Collection
[
|
...
]