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author：knowledge 2025-09-03 11:47:33 - browse：87991

A vial of Tofersen injection on the left of its box — The ALS drug tofersen is marketed as Qalsody in the U.S. Biogen

LONDON — Neurologists and patient advocates are up in arms over a policy decision by a U.K. health agency that they say will imperil access to an ALS treatment that’s available in the U.S. and on its way to approval in the European Union.

The medicine in question, Biogen’s tofersen, is designed to treat a rare genetic form of the neurodegenerative disease, accounting for some 2% of all ALS cases. And the issue is how the U.K.’s cost-effectiveness agency has said it would assess the costs and benefits of the therapy.

The agency, the National Institute for Health and Care Excellence, or NICE, has a special pathway for drugs for very rare diseases, which advocates and doctors argue is the route through which tofersen should be reviewed. But NICE has indicated it would review the drug through its standard process, saying it’s because ALS as a whole does not count as a “very rare” disease. That sets a higher bar to clear.

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