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Otarmeni

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healthcare5 minutes ago•13 min saved

FDA greenlights Otarmeni: first gene therapy to restore hearing in OTOF-related deafness, Regeneron to provide it free in the U.S.

The FDA granted accelerated approval for Otarmeni (lunsotogene parvec-cwha), a one-time gene therapy for severe-to-profound OTOF-related hearing loss, based on CHORD trial results showing 80% of participants reached a key hearing threshold at 24 weeks and 42% achieved normal hearing by 48 weeks; Regeneron will provide Otarmeni free to eligible U.S. patients as part of access programs, with continued confirmatory trials and safety monitoring ongoing.

via Regeneron|

#fda-accelerated-approval #gene-therapy #healthcare

health4 hours ago•13 min saved

First-in-class Otarmeni gene therapy could restore hearing in rare OTOF-related loss, Regeneron to provide free US access

Regeneron’s Otarmeni (lunsotogene parvec-cwha) received FDA accelerated approval as the first in vivo gene therapy for OTOF-related severe-to-profound hearing loss, based on CHORD trial results showing 80% reached a PTA threshold of ≤70 dB HL at 24 weeks and 42% achieved normal hearing (≤25 dB HL) by 48 weeks. The one-time intracochlear infusion is intended for patients with biallelic OTOF variants, preserved outer hair cell function, and no prior cochlear implant in the treated ear, and Regeneron will provide Otarmeni at no cost to eligible U.S. patients. Safety signals include otitis media, dizziness and other procedure-related events; continued approval will depend on confirmatory CHORD data and site-specific eligibility constraints.

via Regeneron|

#fda #gene-therapy #health