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
RELYVRIO (previously known as AMX0035 in the U.S.) is an oral, fixed-dose combination therapy for the treatment of adults with ALS
Verdiperstat, Biohaven’s investigational first-in-class potent, selective, brain-penetrant, irreversible myeloperoxidase (MPO) enzyme inhibitor, failed to statistically differentiate from placebo in newly announced findings from the pivotal HEALEY ALS Platform Trial.
12-month data show earlier initiation of tofersen slowed decline across critical measures of function and strength in people diagnosed with SOD1-ALS
Amylyx Pharmaceuticals announced that the donated clinical data from the placebo arm of the CENTAUR clinical trial that evaluated the safety, efficacy, and survival benefits of AMX0035 in adult participants with amyotrophic lateral sclerosis (ALS) are now available in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database.
On September 7 the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee, comprised of independent advisers to the FDA, voted 7—2 in favor of recommending AMX0035 for approval. The Advisory Committee’s recommendation is not binding. The FDA is expected to decide whether to approve AMX0035 by September 29.
Biogen Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
The HEALEY ALS Platform Trial led by the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in collaboration with the Northeast ALS Consortium (NEALS) is a trial in which multiple investigational products are tested and evaluated simultaneously to accelerate the development of potential new therapies for people with ALS.
Albrioza (formerly AMX0035) was approved by Health Canada for ALS this week. NEALS sites and members were leaders of the CENTAUR trial of Albrioza.
