06/08/2021 at 7:20 PM CEST
For 18 years, Alzheimer’s patients and their families had no news about a possible treatment for this neurodegenerative disease. Yesterday, it was known that Federal Drug Administration (FDA), in charge of giving the go-ahead to drugs in the United States, approved the first drug potentially capable of modifying the course of the disease of Alzheimer’s.
Without a doubt, a momentous and hopeful news, since the last drug against Alzheimer’s disease was approved in 2003, and was intended to alleviate the symptoms of the disease, not the cause of it.
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The specialists of the Spanish Neurology Society (SEN) congratulate themselves on this good news, although they believe that it is necessary to clarify some aspects in this regard.
First, the SEN notes that this authorization is a conditional approval, linked to the FDA’s “Accelerated Approval Pathway”. This pathway is contemplated for ‘drugs against a serious or fatal disease that can provide significant clinical benefit over existing treatments or when the drug has shown an effect on a surrogate marker of efficacy that reasonably predicts a benefit to patients, and when it remains uncertainty about its clinical benefit ”.
The decision has been adopted after intense months of debate and controversy, among other reasons because Biogen (the company that owns the molecule) decided not to complete the studies as planned.
In fact, the FDA itself acknowledges “(evidence) controversial regarding its clinical benefits, (but) the FDA has determined that there is sufficient evidence that Aduhelm (aducanumab) reduces amyloid plaques in the brain, and that such a reduction probably lead to significant benefits for patients. ‘
This type of approval is linked to post-approval studies (phase 4 clinical trials), as has been the case on this occasion, and which contemplates a withdrawal of approval if Biogen it does not provide data confirming the efficacy and safety of the drug in the coming years.
Despite all these conditions, SEN experts believe that “this approval offers hope to the many individuals and families who suffer from this devastating disease. The first message should be one of caution and responsibility to manage the expectations of the families.
Caution necessary for several reasons. The first is that in Spain we must wait for the arrival of this drug. Biogen has applied for approval at the European Medicines Agency (EMA), whose decision is expected in the coming months.
Second, you have to be very clear that this treatment is not a cure for Alzheimer’s disease. Neurologists explain that according to “available data this high-dose drug takes several months to clear amyloid plaques from the brain of patients that have accumulated over years, or even decades.”
Furthermore, according to the information provided by the laboratory that has developed this drug against Alzheimer’s disease, “it would appear that only when a radical reduction of the amyloid load (a practical reversion to normality) is achieved in a sustained manner and in phases initials of the disease could begin to appreciate the clinical benefit of patients, “they detail from the SEN.
This indicates that long studies are necessary to be able to evaluate the efficacy of these drugs. In addition to the data provided by Biogen over the next few years, it is relevant to note that two other drugs with very similar characteristics from pharmaceutical companies Roche (gantenenumab) and Eisai (lecanemab) are conducting phase 3 studies involving several Spanish centers and which will be completed by the end of 2023 and 2024 respectively.
Finally, there are still many open issues such as the cost of the drug, with significant discrepancies between the market price estimate that various media have collected (unaffordable both for a public health system and for an average family economy) and the costs estimated by different sources based on expected effectiveness.
“In any case, experts emphasize, this approval reflects the exciting moment in which we find ourselves in the research and fight against Alzheimer’s and may represent a turning point. It is now possible to modify key pathophysiological processes of the disease with possible clinical benefit. ‘
«Despite the doubts that the FDA approval of AducanumabWe should congratulate ourselves on the enormous advances we have made in the field of Alzheimer’s disease in recent decades, advances so far overshadowed by the repeated failures of clinical trials until yesterday. We hope it will help mobilize more resources in the fight against the disease, from funding agencies and industry.
In short, and although the prospects seem promising, there are still several crucial issues to be resolved. Among them are essential points in relation to the selection criteria, access to the drug, preparation of the health system and cost, in addition to the questions mentioned in relation to efficacy.