FDA aprueba Aduhelm / Biogen, nuevo tratamiento para Alzheimer

 

The Food and Drug Administration on Monday approved the first new treatment for Alzheimer’s disease in nearly two decades, a landmark decision that has been eagerly awaited by millions of Americans diagnosed with the condition but that will be hotly contested by some in the scientific community who doubt the drug’s effectiveness.

While other drugs treat symptoms of Alzheimer’s, the new medicine, called Aduhelm, is the first to attack what some believe is an underlying cause of the disease and slow cognitive decline, albeit marginally. It does so by eliminating clumps of a toxic protein believed to destroy neurons and cause dementia.  

Aduhelm is not a cure for Alzheimer’s, and it doesn’t reverse the disease’s progression.

The drug — which just two years ago was declared a stunning failure — is now expected to generate billions of dollars in revenue for its maker, Biogen.

“This historic moment is the culmination of more than a decade of groundbreaking research in the complex field of Alzheimer’s disease,” Biogen CEO Michel Vounatsos said in a statement. “We believe this first-in-class medicine will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come.”

But the approval of Aduhelm, also known by its scientific name aducanumab, is all but sure to become one of the most controversial and disputed decisions on a drug application in recent years. The FDA granted marketing clearance to the drug over the strong objections of a panel of independent experts it convened in November. Those advisers reviewed Biogen’s clinical data and concluded overwhelmingly that there was insufficient evidence that the treatment had significant benefits for patients. They argued the drug should not be approved, as have many outside experts. Others saw enough evidence of efficacy — and a desperate need for new treatments.

Instead of judging Biogen’s treatment solely on its effects on cognition, the FDA granted a conditional approval based on Aduhelm’s ability to clear the toxic proteins, called beta-amyloid. In order to continue marketing the drug, Biogen will need to complete a large clinical trial to confirm that removing the plaque has cognitive benefits, the FDA said. If that study fails, the FDA has the authority to rescind its approval.

In approving the drug on a conditional basis, the agency departed from decades of regulatory precedent, setting a new bar for treatments with considerable potential but unproven benefits — a standard that could also be applied to other devastating diseases.

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Biogen said the yearly cost for a maintenance dose of Aduhelm, based on an average patient’s weight, would be $56,000. That’s a list price, not the net price or the price paid by patients with insurance. The out-of-pocket cost for patients with insurance will vary depending on their coverage. Analysts had expect it to cost between $10,000 and $25,000 per year, which would have already placed it among the most expensive medicines marketed to primary care physicians. The company expects to start shipping the drug in about two weeks. 

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