FDA seems poised to approve a new drug for ALS, but does it work?

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2022-09-23 06:00:19

But it's still not clear whether the drug, called AMX0035, truly helps people with ALS, a rare and fatal neurological disorder that eventually leaves a person unable to walk, talk, swallow and breathe.

In March, the FDA's Peripheral and Central Nervous System Drugs Advisory Committee concluded that a study of 137 ALS patients did not provide "substantial evidence" that AMX0035 was effective.

Then in September, after prodding from FDA officials and an email campaign by patients and their families, the same committee reconvened, and this time recommended approving the drug.

An approval is more likely now that it would have been decades ago, says Holly Fernandez Lynch, an assistant professor of medical ethics and health policy at the University of Pennsylvania.

AMX0035 is a combination of two existing products. One is a dietary supplement called taurursodiol, which can be purchased online.The other is a prescription drug called sodium phenylbutyrate, which is used to treat a rare type of metabolic disorder.

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