The company will withdraw its amyotrophic lateral sclerosis (ALS) drug – its only product in the market – from the U.S. and Canada after the treatment failed in a key late-stage trial.
Tag Archive for: ALS
Topline data from the Phase III PHOENIX trial of Relyvrio, which won approval in 2022, showed no significant difference on either the primary or secondary endpoints, according to Amylyx.
The past two years have seen a pair of new treatments approved for a particularly intractable neurodegenerative disease —amyotrophic lateral sclerosis. But while Amylyx’s Relyvrio and Biogen’s Qalsody have crossed the FDA finish line, others have stumbled in a regulatory space that experts say is still evolving.
The agency found data from the company’s mid-stage study for the treatment of a type of neurodegenerative disease to be insufficient to support accelerated approval.
The decision comes after Amylyx in June requested a formal re-examination of the initial negative opinion adopted by the Committee for Medicinal Products for Human Use of the European Medicines Agency.
The Japanese multinational pharma is pledging up to $580 million in a development and commercialization deal with AcuraStem for the latter’s PIKFYVE program for amyotrophic lateral sclerosis.
The study will use a robot to surgically place a brain-computer interface (BCI) implant in a region of the brain that controls the intention to move, Neuralink said, adding that its initial goal is to enable people to control a computer cursor or keyboard using their thoughts alone.
After its Biologics License Application was rejected twice by the FDA, BrainStorm’s Phase III data suggest its amyotrophic lateral sclerosis candidate significantly lowers neurofilament light chain levels.
The Massachusetts-based biopharma became the second company this week to drop its amyotrophic lateral sclerosis hopeful after it failed to elicit significant improvement compared with placebo.
The company said the decision followed a review of a late-stage study by a data monitoring panel, which showed the drug, reldesemtiv, had no effect in ALS patients compared with placebo.
