Un articolo di STAT riporta nuove rivelazioni sulla discussa approvazione da parte di FDA del farmaco educanumab (nome commerciale Aduhelm), un trattamento a base di anticorpi monoclonali anti-beta amiloide. Aduhelm verrà commercializzato come farmaco contro l’Alzheimer nonostante gli studi clinici di terza fase non abbiamo dimostrato in modo inequivocabile la sua efficacia. L’approvazione, infatti, si basa solamente sul provato effetto del farmaco sulla riduzione delle placche amiloidi, legate al morbo di Alzheimer. Per giustificare l’approvazione di questo farmaco, nonostante il parere negativo di un gruppo di consulenti esterni, FDA ha utilizzato una tipologia di approvazione di emergenza, accelerated approval, mai utilizzata prima in simili situazioni:
Accelerated approval is traditionally used for treatments that haven’t yet proved themselves in large trials. In Biogen’s case, Aduhelm went through two Phase 3 studies and came up with conflicting evidence. But the council, citing federal law, concluded that the FDA had the authority to use accelerated approval on any treatment “upon a determination that the product has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.” Amyloid fit the bill.
“We recognized this actually fit very well into that accelerated approval paradigm,” said Peter Stein, director of the FDA’s Office of New Drugs, in a Tuesday interview with STAT. “This was not us trying to fit a round peg into a square hole. This is a program that fit very well.”
FDA officials acknowledged the accelerated approval is unusual because it is usually used when studies assessing efficacy are not completed. In the case of Aduhelm, the issue is that one of these studies had failed.
La tortuosa approvazione di questo farmaco e le sue potenziali conseguenze sulla ricerca per nuovi farmaci contro l’Alzheimer sono discusse anche in un episodio del podcast NPR Short-Wave:
One question is whether pharmaceutical companies that developed and then gave up on other amyloid drugs will be asking the FDA to approve these drugs the same way it did aducanumab. And ironically, these companies may only have to show that their drug is as effective as aducanumab, a drug that maybe doesn’t even work. But the future of Alzheimer’s drugs is probably going to be about targets other than amyloid. For example, right now, there are a number of experimental drugs that target a protein called tau. So a lot of researchers think there are going to be much more effective treatments for Alzheimer’s in the next few years. They just won’t target amyloid.
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