Otsuka heads to FDA as Ionis-partnered ALS drug hits primary phase 3 endpoint

Otsuka heads to FDA as Ionis-partnered ALS drug hits primary phase 3 endpoint — Live Feedhttps://www.live-feeds.com/feed/otsuka-heads-to-fda-as-ionis-partnered-als-drug-hits-primary-phase-3-endpointContinuously updated, source-cited coverage. Otsuka and Ionis move ulefnersen toward FDA approval after Phase 3 successhttps://www.live-feeds.com/feed/otsuka-heads-to-fda-as-ionis-partnered-als-drug-hits-primary-phase-3-endpointhttps://www.live-feeds.com/feed/otsuka-heads-to-fda-as-ionis-partnered-als-drug-hits-primary-phase-3-endpoint#u83133Fri, 25 Sep 2026 00:06:16 +0000Otsuka is preparing an FDA submission for ulefnersen following positive topline results from the Phase 3 FUSION study. The drug met its primary endpoint and demonstrated a favorable safety and tolerability profile. Developed in partnership with Ionis Pharmaceuticals, ulefnersen is positioned as the first potential disease modifying treatment for FUS-ALS. To provide immediate options for patients, Otsuka has established an Early Access Program for eligible individuals living with FUS-ALS while the formal regulatory process proceeds.Why it mattersFUS-ALS is a specific form of amyotrophic lateral