Biogen Publishes P-III (VALOR) Study Results of Tofersen for the Treatment of Amyotrophic Lateral Sclerosis at NEJM


The company reported the results from the P-III (VALOR) study in 108 patients & OLE study in 95 patients evaluating the effects of tofersen (100mg) for ALS associated with a SOD1 mutation
The results showed a sustained reduction in SOD1 protein & neurofilament. The results also slowed the decline in clinical function, respiratory function, strength, and QoL with earlier initiation of tofersen
The US FDA has accepted the tofersen’s NDA for priority review & the PDUFA date is expected on Jan 25, 2023. The therapy is being evaluated in the P-III (ATLAS) study to evaluate tofersen & delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity

Ref: Biogen | Image: Biogen