F.D.A. Approves Alzheimer's Drug Despite Fierce Debate Over Whether It Works - The New ... - 0 views
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The Food and Drug Administration on Monday approved the first new medication for Alzheimer’s disease in nearly two decades, a contentious decision, made despite opposition from the agency’s independent advisory committee and some Alzheimer’s experts who said there was not enough evidence that the drug can help patients.
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The drug, aducanumab, which go by the brand name Aduhelm, is a monthly intravenous infusion intended to slow cognitive decline in people in the early stages of the disease, with mild memory and thinking problems. It is the first approved treatment to attack the disease process of Alzheimer’s instead of just addressing dementia symptoms.
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During the several years it could take for that trial to be concluded, the drug will be available to patients, the agency said. If the post-market study, called a Phase 4 trial, fails to show the drug is effective, the F.D.A. can — but is not required to — rescind its approval.
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But the F.D.A. advisory committee, along with an independent think tank and several prominent experts — including some Alzheimer’s doctors who worked on the aducanumab clinical trials — said the evidence raised significant doubts about whether the drug is effective.
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The F.D.A. authorized the drug under a program called accelerated approval, which has been applied to therapies for some cancers and other serious diseases for which there are few, if any, treatments.
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The risks with aducanumab involve brain swelling or bleeding experienced by about 40 percent of Phase 3 trial participants receiving the high dose. Most were either asymptomatic or had headaches, dizziness or nausea. But such effects prompted 6 percent of high-dose recipients to discontinue. No Phase 3 participants died from the effects, but one safety trial participant did.
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About two million Americans may fit the description of the patients the drug was tested on: people in the early stages of Alzheimer’s or the stage just before that, Alzheimer’s-related mild cognitive impairment. About six million people in the United States and roughly 30 million globally have Alzheimer’s, a number expected to double by 2050. Currently, five medications approved in the United States can delay cognitive decline for several months in various Alzheimer’s stages.
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The crux of the controversy over aducanumab involved two Phase 3 trials with results that contradicted each other: One suggested the drug slightly slowed cognitive decline while the other trial showed no benefit. The trials were stopped early by a data monitoring committee that found aducanumab didn’t appear to be showing any benefit. Consequently, over a third of the 3,285 participants in those trials were never able to complete them.
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Aducanumab, a monoclonal antibody, targets a protein, amyloid, that clumps into plaques in the brains of Alzheimer’s patients and is considered a biomarker of the disease. One thing both critics and supporters of approval agree on is that the drug substantially reduces levels of amyloid, and the F.D.A. said that the drug’s effect on a biomarker qualified it for the accelerated approval program.
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He estimates 25 to 40 percent of the clinic’s roughly 3,000 patients might be eligible, but it doesn’t have enough neurologists.