Biogen Seeking A Path Forward After Phase III Tofersen Study Fails In ALS
Ionis-Partnered Asset Developed For SOD1 Genetic Mutations
The drug showed favorable but not significant trends across secondary endpoints, and greater efficacy in slower-progressing patients and those treated earlier in the disease, in a small subpopulation.
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After the Aduhelm controversy, Biogen has another neurodegenerative disease drug which so far can only show efficacy against surrogate biomarkers – will the FDA be swayed again this time?
Company seeks accelerated approval based on changes in neurofilament as a surrogate for efficacy; with controversy over FDA’s Aduhelm approval in Alzheimer’s still raging, agency is again faced with deciding whether biomarker data in a neurodegenerative disease with high unmet need are sufficient to justify approval in the face of a failed clinical endpoint study.
FDA accepted the NDA for tofersen based on a surrogate biomarker after a Phase III trial failure, echoing some elements of the Aduhelm experience, though in this instance the target patient population is ultra-niche.