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Here We Go Again: US FDA Weighing Accelerated Approval For Biogen’s ALS Drug Tofersen

 
• By Sue Sutter

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.

Opening the door
Tofersen could open the door to use of the accelerated approval pathway for other ALS treatments. • Source: Shutterstock

With Biogen, Inc.’s tofersen now under review for a subgroup of ALS patients, the US Food and Drug Administration is once again faced with deciding whether biomarker data in a neurodegenerative disease with high unmet need justify accelerated approval in the face of a failed clinical endpoint study.

On 26 July, Biogen announced the FDA had accepted its application for tofersen, an antisense oligonucleotide, for treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The NDA is undergoing a priority review with a user fee goal date of 25 January 2023

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