lack of efficacy

FDA advisory committee votes against NurOwn approval for ALS

In a nearly unanimous vote, an advisory committee to the U.S. Food and Drug Administration (FDA) said there’s not enough evidence to support the efficacy of the experimental stem cell therapy NurOwn as a treatment for amyotrophic lateral sclerosis (ALS). The committee voted on a single question in its meeting: “Do…

UCB Stops Zilucoplan Arm Early in HEALEY Trial Due to Lack of Benefit

The Phase 2/3 clinical trial testing UCB’s investigational therapy zilucoplan for amyotrophic lateral sclerosis (ALS) — one of the arms of the multi-regimen HEALEY ALS platform trial — has been stopped early based on interim data. The decision was made after a pre-specified analysis “demonstrated futility,” UCB said…

Dimethyl Fumarate (DMF) Fails to Slow ALS Progression in Trial

Dimethyl fumarate or DMF — an oral therapy approved for multiple sclerosis (MS) — was well-tolerated among adults with amyotrophic lateral sclerosis (ALS) but failed to significantly slow disease progression or to improve survival and quality of life in these patients, according to data from a Phase 2…