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Motor neuron disease treatment misses primary endpoint but reports benefits within a year

BMJ 2022; 378 doi: (Published 23 September 2022) Cite this as: BMJ 2022;378:o2301
  1. Elisabeth Mahase
  1. The BMJ

Antisense drug tofersen may slow and reduce disease progression in patients with motor neuron disease (MND) caused by the faulty SOD1 gene, according to the results of a phase 3 clinical trial.1

Researchers from the Sheffield Institute for Translational Neuroscience (SITraN) compared changes in physical functioning between 108 participants that were given eight doses of 100 mg tofersen (72 people) or placebo (36) over a period of 24 weeks.

The trial, funded by the biotechnology company Biogen, missed its primary endpoint of significant clinical improvement at 28 weeks and was then extended to 52 weeks, at which point the researchers said there were notable changes in motor and lung function.

Despite failure to meet the primary endpoint, Biogen has already submitted the drug to the US Food and Drug Administration (FDA) for accelerated approval. The FDA has granted a priority review for the application, with a decision expected …

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