Rare genetic diseases stories

Sep 22, 2026

Otsuka and Ionis' ulefnersen meets primary endpoint in phase 3 ALS trial

Otsuka Pharmaceutical and Ionis Pharmaceuticals announced their experimental drug ulefnersen met the main goal of a late-stage study in patients with a rare inherited form of amyotrophic lateral sclerosis (ALS), showing improvements in functional impairment and survival. The partners plan to approach the FDA to determine potential regulatory pathways.

Sep 22, 20266 sources

Top claims

▪Ulefnersen expands Ionis Pharmaceuticals' genetic amyotrophic lateral sclerosis portfolio, which also includes Qalsody, an FDA-approved drug targeting SOD1-related ALS developed in partnership with Biogen.
▪The Phase 3 FUSION trial of ulefnersen met several secondary endpoints, including reducing serum neurofilament light chain levels and delaying time to death, permanent ventilation, rescue, or disease-related withdrawal.
▪Otsuka Pharmaceutical plans to discuss the Phase 3 FUSION trial results with the U.S. Food and Drug Administration and other global health authorities to explore potential expedited regulatory approval pathways for ulefnersen.

Subtopics

Clinical trials1 Drug Approval and Clinical Trials1

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