FDA approves new Alzheimer’s treatment despite risks, unclear benefits

A new Alzheimer's disease treatment that may slightly slow the progression of cognitive decline in the disease's early stages was granted a fast-tracked approval by the Food and Drug Administration on Friday.

The treatment, called lecanemab, is made by pharmaceutical companies and is used to treat Alzheimer's disease and other dementias. If amyloid plaques are a root cause of the disease, researchers don't know if clearing them can slow or stop cognitive decline.

A surrogate endpoint that is reasonably likely to predict a clinical benefit to patients is what the FDA used to approve lecanemab. Lecanemab's ability to reduce amyloid plaques in the brains of Alzheimer's patients was the surrogate endpoint.

Uncertain efficacy

The significance of that and the drug's effectiveness are not certain. The New England Journal of Medicine published a study showing that lecanemab slowed cognitive decline in patients with early Alzheimer's. 898 people were assigned to receive lecanemab and 971 people were assigned a placebo. An established clinical test for dementia was used to assess their cognitive abilities. The lecanemab treatment group's score fell by 1.21 points after 18 months, while the placebo group's fell by 1.66 points.

It's difficult to say if that change is significant. According to a scientist, the drug's ability to clear amyloid plaques was exciting. The difference between lecanemab and placebo is not considered to be a clinically meaningful treatment effect.

Longer trials are needed to determine the efficacy and safety of lecanemab in early Alzheimer's disease.

Safety concerns

There are reports that three patients have died from brain swelling and bleeding after receiving the drug. A 65-year-old woman died of a massive brain hemorrhage. Castellani told Science in November that he believed the drug weakened the woman's blood vessels, which then burst from a common treatment for blood clot.

Castellani said it was a one-two punch. I have no doubt that this is a treatment-caused illness and death. The patient would be dead today if she hadn't been on lecanemab.

The woman's death was reported in the New England Journal of Medicine.

There are warnings about the use of blood thinners and brain bleeding.

The FDA's approval comes just a week after lawmakers released the results of an 18-month congressional investigation into the agency's approval of Aduhelm. The FDA granted approval for that therapy despite the fact that it was less conclusive than lecanamab.

Irregularities

The FDA's approval process was found to berife with irregularity and inappropriate communications between the FDA and Biogen. The report criticized Biogen for setting a high price for Aduhelm.

The report documents the atypical FDA review process and corporate greed that preceded FDA's controversial decision to grant accelerated approval to Aduhelm.

I am hopeful that lecanemab will live up to its promise of slowing the progression of Alzheimer's disease for patients and their loved ones. I hope the price of lecanemab will be fair to ensure patients have equitable access to the drug.

The price of lecanemab was announced in a press release Friday afternoon. That is above the range that the Institute for Clinical and Economic Review estimated would make the drug cost-effective.

It is not clear if Medicare will cover lecanemab. Due to the high price and lack of evidence of benefit, Medicare limited coverage of Aduhelm. Only Medicare beneficiaries in clinical trials are covered for the price of Aduhelm.