FDA Advisory Committees provide non-binding recommendations for consideration by the FDA. The New Drug Application for tofersen for the treatment of SOD1-ALS was submitted to the FDA for consideration under accelerated approval. The FDA is continuing its review of tofersen with a Prescription Drug User Fee Act action date of April 25, 2023.
Click here to watch a recording of The ALS Association webinar on Tofersen and the FDA Approval Process.
Amylyx anticipates topline results in 2024. The study enrolled 664 participants living with ALS.
Serum and cerebrospinal fluid samples from the Phase 3 study of NurOwn® in ALS to be donated to the NEALS biorepository for use by the research community
The HEALEY ALS Platform Trial is adding a new investigational drug, DNL343, to continue to research new treatments for ALS (Amyotrophic Lateral Sclerosis or Lou Gehrig’s disease).
The Annual Meeting of the Northeast Amyotrophic Lateral Sclerosis (NEALS) Consortium brings together clinicians, researchers, and members of the NEALS Consortium to share the latest data and findings in the care of ALS and discuss the hottest topics in neuromuscular medicine.
Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of an interim analysis of the Phase 4 REFINE-ALS study, designed to identify predictive and pharmacodynamic biomarkers to measure the effect of RADICAVA® (edaravone) in people with amyotrophic lateral sclerosis (ALS).
We are thrilled to report that this year’s abstracts will be published electronically in Muscle and Nerve.
In an open-label extension trial, tofersen showed significant reductions in SOD1 protein and neurofilament light over a 12-month period. It is now expected to be reviewed by the FDA by early Q2 2023.
A secondary endpoint analysis of survival demonstrated a significant reduction in risk of death or permanently assisted ventilation when adjusting for baseline risk imbalances in the CNM-Au8 regimen for the 30 mg dose at 24 weeks
