The US drug agency approved a medication to treat this disease in its initial stage. Groups of patients demanded his authorization, although there are medical experts who object to clinical trials
After several twists and turns, the United States drug regulatory agency, known as the FDA , announced today thatfast-track authorization of the use of a new drug to treat early-stage Alzheimer’s disease, but asked the Biogen laboratory – which leads the development – to do more research to have more data on its effectiveness.
The drug is called aducanumab. Its trade name is Aduhelm. Patient and family organizations had called for research and approval of more effective treatments, such as aducanumab, but dementia experts have raised objections. Those experts disagree on the way in which clinical trials were carried out and point out that there is still uncertainty about its true efficacy in counteracting the disease.
Drug approval comes with a caveat, Because the FDA requires that Biogen conduct an additional placebo-controlled study of the drug to verify its effectiveness in improving people’s memory and cognitive symptoms.
That request stems in large part from the conclusion of an expert panel that decided almost unanimously thatthe evidence presented by the drug’s developers did not justify approval.
The drug was developed by the Biogen laboratory in collaboration with Eisai. The company had finished two phase 3 trials early, saying the drug had failed. However , after reexamining their data, the company found that in one of those studies, patients who received a high dose of aducanumab for a long period of time benefited . Having these data, he submitted the request for the FDA to approve the drug.
As reported by Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research of the US regulatory agency, the use of Aduhelm (aducanumab) has been approved for the treatment of patients with Alzheimer’s disease using the accelerated approval pathway. This mechanism was used when considering that it is “a drug for a serious or life-threatening disease that can provide a significant therapeutic benefit over existing treatments when the drug is shown to have an effect on a surrogate endpoint that has a reasonable probability of predicting clinical benefit for patients and there remains some uncertainty about the clinical benefit of the drug.
In the press release, Dr. Cavazzoni argued: “ This approval is significant in many ways. Aduhelm is the first novel therapy approved for Alzheimer’s disease since 2003 . Most significantly, Aduhelm is the first treatment to target the underlying pathophysiology of Alzheimer’s disease, the presence of beta amyloid plaques in the brain. Aduhelm’s clinical trials were the first to show that reducing these plaques – a hallmark finding in the brains of Alzheimer’s patients – is expected to lead to a reduction in the clinical deterioration of this devastating form of dementia. “
The drug would be indicated for people with Alzheimer’s in the initial phase. It could have an impact on millions of people with the disease in the US. Today’s approval will also be a key decision for regulatory agencies in other countries to also approve.So far the treatments that are indicated for Alzheimer’s only treat the symptoms.
Alzheimer’s is a progressive neurological disease that affects the thinking and independence of millions of people around the world. Currently, more than 40 million people around the world are living with Alzheimer’s. But it is also the third leading cause of death in people over 65, after cancer and cardiovascular disease.
Generally, Alzheimer’s disease is most commonly diagnosed in people over the age of 65, but it begins earlier with subtle neurological changes that occur over years or even decades before symptoms appear. Many people experience the often unidentified early signs of mild cognitive impairment.
In 1987, the first clinical trial on the disease was launched. Since 1998, 100 drugs have been studied, including a vaccine in mice. But only 5 drugs have been authorizedThey can help control some of the symptoms. There are numerous treatments and diagnostic tests that are under investigation and are in various stages of clinical trials.
The research and development of better treatments to stop, slow down or even prevent it has become a priority for many scientists in the world considering that by the year 2030 the number of people with Alzheimer’s could reach 65 million and by 2050, it could exceed the 100 million.
Last November, an FDA advisory committee voted by majority against recommending the approval of aducanumab. They argued that the data did not convincingly show that the drug slowed cognitive decline. Later, three members of the advisory committee wrote a point-by-point critique of the tests. Other scientists, and a group of independent experts, say that aducanumab has not shown a convincing benefit that outweighs its safety risks.
Beyond the status of the Alzheimer’s drug in particular, some experts are concerned that approval could lower standards for other drugs in the future, an issue especially important at a time when public trust in science is faltering.
One of the Alzheimer’s researchers, who has participated in conducting clinical trials for different companies, Jason Karlawish , professor of medicine, ethics and public health policy at the University of Pennsylvania, United States, wrote that he has been answering patients for 18 years and relatives that there is nothing new for Alzheimer’s.
“The few drug treatments that I prescribe are only modestly effective in alleviating the cognitive problems of the disease,” he wrote days ago in Stat . And he anticipated that he would not recommend the drug aducanumab to his patients if approved by the FDA. The cost of the drug would be between $ 20,000 and $ 50,000 per person per year.
“I think the company is wrong, and I agree with the analysis of the Institute for Clinical and Economic Review; the data to argue this case are murky and, even if they were clear, the benefits of the drug are ambiguous at best and do not deserve this cost, “he said.
In addition, Karlawish considered “if aducanumab is approved, patients and their families will have to fight to know if it is right for them. One of its risks is small brain hemorrhages, a risk that is accentuated in those who have the APOE4 gene, a gene associated with late-onset Alzheimer’s disease. Families will be drawn into these discussions of risks and benefits, as treating a parent’s Alzheimer’s with aducanumab may mean that their children are aware of their genetic risk of developing the disease. ” It was of the opinion that it has not been adequately studied, so the FDA has incomplete data to make a judgment.
Following today’s approval, the FDA director acknowledged that there was debate about the drug. But he came out to defend the decision. “Aduhelm’s development program in its final phase consisted of two phase 3 clinical trials. One study met the primary endpoint, showing a reduction in clinical deterioration. The second trial did not meet the primary endpoint. However, in all the studies in which it was evaluated, Aduhelm consistently and very convincingly reduced the level of amyloid plaques in the brain in a dose- and time-dependent manner. The reduction in amyloid plaque is expected to translate into a reduction in clinical deterioration.
The FDA advised that it will continue to monitor Aduhelm as it reaches the market and, ultimately, at the bedside. Additionally, it required Biogen to conduct a post-approval clinical trial to verify the drug’s clinical benefit. “ If the drug does not work as intended, we can take steps to withdraw it from the market. But hopefully we will see more evidence of benefit in the clinical trial and as more people receive Aduhelm. As an agency, we will also continue working to promote the development of drugs for this catastrophic disease, “they concluded.