Drug for Rare Form of ALS Earns FDA Nod
The FDA granted accelerated approval to tofersen (Qalsody) for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, the agency announced on Tuesday. The drug was cleared under the accelerated approval pathway, a program that uses a surrogate endpoint to approve drugs for serious conditions with an unmet medical need. For tofersen, the […]
Investigational ALS Drug May Have Clinical Benefit, FDA Staff Says
Tofersen, an investigational antisense drug developed to treat amyotrophic lateral sclerosis (ALS) associated with a mutation in the SOD1 gene (SOD1-ALS), may have clinical benefit, FDA reviewers indicated in briefing documents ahead of an advisory committee meeting. On Wednesday, the Peripheral and Central Nervous System Drugs Advisory Committee will vote on whether evidence supporting a […]
Investigational ALS Drug Betters Biomarkers, but Can It Improve Motor Functions?
Tofersen, an investigational antisense oligonucleotide developed to treat an inherited form of amyotrophic lateral sclerosis (ALS), changed biomarkers and curbed neurodegeneration but did not improve motor control and muscle strength at 6 months, the phase III VALOR trial showed. In a subgroup of patients with fast-progressing SOD1 ALS, the 28-week change in scores on the […]