A drug that is effective in Alzheimer's disease? There can be no doubt that such a drug would be more than welcome for a large number of patients, their families and friends, and healthcare professionals. And yet, the announcement by the US Food and Drug Administration (FDA) that marketing authorisation (MA) had been granted for what it called "the first therapy to target and affect the underlying disease process of Alzheimer's" has been met with much criticism.

After failing to demonstrate efficacy in terms of disease progression, the company discontinued the two initial phase 3 clinical trials. However, it then reanalysed the data and submitted a request for MA to the FDA, based primarily on a re- duction in amyloid plaques. In 2020, the FDA's advisory committee for nervous system diseases issued a nearly unanimous negative opinion regarding this request. Yet the FDA nevertheless granted an accelerated MA for all patients, supported by the reduction in amyloid plaques, a non-clinical endpoint which has not been shown to be linked to disease progression in numerous studies. In addition, the FDA has given the company 9 years in which to carry out another comparative trial using clinical endpoints.

This MA has sparked much criticism and led to the resignation of three members of the expert advisory committee. With a treatment price of 56 000 dollars per year, the company will certainly find ways to fuel demand for this drug between now and 2030, through investment in advertising in the press and publicity aimed at affected individuals, as well as financial incentives to prescribing doctors, and the funding of continuing medical "education".

©Prescrire 1 January 2022

Source: "Alzheimer's: yet another unwelcome drug" Prescrire International 2022; 31 (233): 26. Free.

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