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FDA approves new ALS medicine in precedent-setting decision

April 25, 2023


The Food and Drug Administration has conditionally approved a new ALS medicine in a decision likely to influence how other experimental treatments for the nerve-destroying disease are tested and reviewed.

The medicine, known until now as tofersen, is only for ALS patients who have a specific genetic mutation. Estimates cited by the FDA hold that this group accounts for less than 500 of the roughly 30,000 people in the U.S with the disease.

Until Tuesday, the few therapies that had secured FDA approval did so because they were shown to help patients live a bit better or a bit longer. Tofersen, which will be sold as Qalsody, is different.

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