The US Food and Drug Administration (FDA) has approved the use of Aduhelm based on the antibody aducanumab. A drug made as a treatment against the possible causes of the disease. The decision has caused a stir because it was made by the US regulator despite the fact that a committee of FDA experts indicated in 2020 that there was insufficient evidence to support the effectiveness of the product.
In a statement issued For the FDA, the Food and Drug Administration of the United States, Patrizia Cavazzoni, director of the Center for Evaluation and Research of Drugs of the body, said that current treatments for Alzheimer’s only attack its symptoms.
“This treatment option is the first therapy to target and affect the underlying Alzheimer’s disease process. As we have learned from the fight against cancer, the expedited approval pathway can get therapies to patients quickly and at the same time. stimulate more research and innovation, “the statement read.
The text, issued on June 7, indicates that the last drug approved to treat symptoms related to the disease was memantine, whose authorization was received almost two decades ago.
The new product, made by the pharmaceutical company Biogen and its Japanese partner Eisai, is structured on the basis of aducanumab, an antibody that supports a molecule obtained from a lucid old man and will receive the trade name of Aduhelm.
This drug acts on beta amyloid proteins, the accumulation of which in cells could be linked to cognitive decline and memory loss. Prior to its approval, this drug was tested in three different clinical trials, involving 3,500 patients.
The results were confusing. In one of the trials, in which 1,600 patients participated, a clinical improvement of 23% was observed while another, with a similar sample, did not show positive results.
“The data supports patients and caregivers who have the option of using this drug … The FDA carefully weighed the needs of people living with the devastating, debilitating and deadly disease,” Cavazzoni told reporters.
The drug received approval under the condition that those who administer it must be part of what is called a phase 4 trial, meaning that researchers will be monitoring patients to assess progress with the treatment and see if it really slows progression of mental deterioration.
Approved by the FDA, applauded by some and questioned by others
The announcement of the drug’s approval by the US regulatory body fueled long-standing debates about the standards used to evaluate therapies in difficult-to-treat conditions.
However, groups representing Alzheimer’s patients and their families applauded the decision. For years, these groups have argued that any new therapy, even of small benefit, deserves approval.
According to the Spanish newspaper El País, which cites groups affected by the disease such as the Alzheimer’s Association of the United States, it indicates that in recent months the authorities have received pressure to approve aducanumab.
“While the trial data has raised some questions in the scientific community, this must be balanced against the certainty of what this disease will do to millions of Americans in the absence of treatment,” wrote Joanne Pike, spokeswoman for the Asociation.
However, the controversy over its approval is that, for a short period of time, the product has not shown positive results.
Additionally, in November 2020, an independent group of experts, convened by the FDA itself, questioned the effectiveness of the drug. The committee, almost unanimously, voted no to a series of questions as to whether the data analyzed from a single study presented by Biogen showed it to be effective.
A year earlier, in March 2019, the pharmaceutical company Biogen announced that it had stopped two studies after obtaining disappointing results. These suggested that aducanumab did not meet its goal of slowing mental and functional decline in Alzheimer’s patients.
However, in a turnaround shown months later, Biogen scientists announced that the initial failure was due to some patients not receiving high enough doses to delay the disease. On this, the FDA warned that said analysis required a review.
Regarding approval, the AP news agency notes that experts warn that giving the drug the green light could set a dangerous precedent, opening the door to treatments of dubious benefit.
“I am surprised and disappointed by the decision … the FDA gets the respect it has because it has regulatory standards that are based on firm evidence. In this case, I think they gave the product a pass,” said Advisor Dr. Caleb Alexander. from the FDA and a medical researcher at Johns Hopkins University.
On the other hand, the doctors expressed doubts about the changes in the position of the regulator in such a short time.
“It is very doubtful that this decision will put an end to the great controversy generated in the scientific community regarding whether sufficient scientific evidence has been presented or whether the FDA has succumbed to the various pressure groups and the urgent need for useful tools to tackle this devastating disease “, declared the biochemist Miguel Medina, deputy scientific director of the Center for Biomedical Research in Network on Neurodegenerative Diseases in Madrid, quoted by the Spanish media.
Product price, another point for controversy
Another point regarding the debate centers on the price of a year of treatment. Biogen announced that the drug would cost approximately $ 56,000 for one year and that it would not increase for another four years.
On this, the non-profit organization Institute for Clinical and Economic Review, quoted by the AP news agency, said that “any price is too high”, if the benefit of the drug is not confirmed in follow-up studies.
According to the World Health Organization (WHO), there are at least 35 million people affected by Alzheimer’s disease in the world. Of that number, at least 6.2 million are Americans according to the FDA statement.
Currently, according to the AP, some 600 doctors in the United States already prescribe the product based on the results of Biogen. This laboratory announced in October that the European Medicines Agency (EMA) is reviewing the application for authorization for the marketing of aducanumab in the European Union.
With Reuters, EFE and AP