The U.S. Food and Drug Administration has agreed to review Biogen Inc's experimental treatment for an inherited form of amyotrophic lateral sclerosis (ALS), after the therapy failed a late-stage study but showed promise in some patients.
The drug, tofersen, in October failed to produce a statistically significant change in functional status for patients with fast-progressing ALS compared to placebo after 28 weeks of treatment.
In June, the company said data from a follow-on study suggested that early administration of tofersen can benefit people living with ALS who have a genetic mutation called SOD1.
It is a type of mutation that leads to production of more of SOD1 protein that can accumulate to toxic levels in ALS patients with mutations in a specific gene.
Biogen is seeking approval for tofersen under the FDA's accelerated approval pathway for treatment of ALS patients. The same pathway was used by the company for its controversial Alzheimer's Disease drug, Aduhelm, last year.
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