WebMar 23, 2024 · Tofersen’s target action date is April 25. Licensed from Ionis in 2024, tofersen is an investigational antisense oligonucleotide that works by targeting the mutant SOD1 mRNA and lowering the levels of the faulty protein. The FDA accepted Biogen's New Drug Application for tofersen in July 2024. WebApr 13, 2024 · 如果FDA批准了tofersen，这将是ALS药物领域第一次基于“生物标志物”数据获批上市。 FDA的最终决定应该不迟于4月25日，预计将影响 ALS研发者如何设计他们 …

FDA advisers support conditional clearance of Biogen’s ALS drug

WebJul 27, 2024 · Tofersen is an RNA-based therapy designed to lower production of the abnormal and toxic SOD1 protein, which is made when the SOD1 gene is mutated. Mutations in this gene are found in up to 20% of people with familial ALS, and up to 2% of those with sporadic disease. WebMar 23, 2024 · 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 ... effect of metoprolol on stress test

Advisory Committees FDA

WebApr 23, 2024 · History of systemic hypersensitivity reaction to tofersen, the excipients contained in the formulation, and if appropriate, any diagnostic agents to be administered during the study. ... Studies a U.S. FDA-regulated Drug Product: Yes: Studies a U.S. FDA-regulated Device Product: No: Additional relevant MeSH terms: Layout table for MeSH … http://www.yyjjb.com.cn/yyjjb/202404/202404101225402540_15058.shtml WebThis week, Jeremy is joined by Larry Falivena, a member of The ALS Association’s Board of Trustees and Dr. Kuldip Dave, vice president of research at The ALS Association, for reaction to the decision by an FDA advisory committee to clear the path for approval of tofersen – and why it’s important for… effect of milk adulteration on human health