US approves first gene therapy for rare form of hearing loss

WASHINGTON, United States, April 24 (AFP): US health officials on Thursday greenlit a first-of-its-kind gene therapy to treat a rare form of hereditary hearing loss, a breakthrough which could pave the way for other such hearing impairment treatments.

Two to three of every 1,000 children in the United States are born with a hearing impairment, and it is estimated that more than half of these cases of early-onset hearing loss are caused by genetic mutations.

Developed by the American biotechnology company Regeneron, the treatment known as Otarmeni targets a rare form of hearing loss which affects around 50 newborns a year in the US.

It will be available for children and adults with severe-to-profound hearing loss caused by certain mutations in the OTOF gene, which encodes a protein critical for transmitting auditory signals from the inner ear to the brain.

While gene therapies are generally extremely expensive -- particularly in the United States, where they can cost several million dollars per patient -- Regeneron said it intends to offer this treatment free of charge to eligible American patients.