On Tuesday 7 June, the FDA (Food and Drug Administration) – the US government body that deals with the regulation of food and pharmaceutical products – approved the first treatment in the world that seems to be able to slow down the degenerative course of the disease of Alzheimer. It’s a matter of Aduhelm – produced by the American biotechnology multinational Biogen – a drug which aims at the fundamental pathophysiology of the disease by preventing its progression. An epochal turning point that, after about twenty years of research, could provide a significant therapeutic advantage over existing treatments.
What is Alzheimer’s disease
L’Alzheimer it is a progressive brain disease that slowly takes root in memory by altering thinking skills and ultimately inhibiting the ability to perform simple routine activities. Although the specific causes of the disease are not fully known, we know that it is characterized by changes in the brain, including so-called “amyloid plaques” and “neurofibrillary tangles” that result in the loss of neurons and their connections. These changes affect a person’s ability to remember and think.
“Alzheimer’s disease is a devastating disease that can have a profound impact on the lives of people diagnosed with the disease and their loved ones“explained Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research.”Currently available therapies treat only the symptoms of the disease; – he then specified –this treatment option is the first therapy to target and influence the underlying disease process of Alzheimer’s. As we’ve learned from the fight against cancer, the fast-track approval path can bring therapies to patients faster, while stimulating more research and innovation.“.
The new drug
It is the first approved treatment for Alzheimer’s since 2003 and also the first therapy that targets the underlying pathophysiology of the disease. The drug consists of a therapy with monoclonal antibodies which can be injected intravenously in patients with moderate Alzheimer’s disease. The substances competing with the composition of the medicine bind to molecules present in the amyloid plaques (whose accumulation in the brain is associated with the disease, ed), thus reducing the pathology run and allowing people to continue carrying out daily activities such as cleaning the house and doing shopping.
Doubts about the treatment
The information contained in the prescription Aduhelm’s – the FDA website reads – include a warning for amyloid-related imaging abnormalities (ARIA), which most commonly presents as temporary swelling in areas of the brain that usually resolves over time and causes no symptoms , although some people may experience headache, confusion, dizziness, vision changes or nausea. Another warning for Aduhelm is the risk of allergic reactions, including angioedema and hives. The most common side effects of Aduhelm are: headache, fall, diarrhea and confusion, delirium, altered mental status and disorientation.
Under the accelerated approval provisions, which provide patients with the disease with early access to treatment, the FDA has requested the company Biogen to conduct a new randomized, controlled trial to verify the clinical benefit of the drug. If the observation fails to substantiate the clinical benefit of the drug, the FDA may initiate a process to withdraw the approval.
Biogen did not disclose the price of Adhulem but analysts estimated that the drug could cost between $ 30,000 and $ 50,000 for one year of treatment. A preliminary analysis by a group found that the drug would need a price of between $ 2,500 and $ 8,300 per year to be well priced for the results. The Institute for Clinical and Economic Review added that “any price is too high“if the benefit of the drug is not confirmed in the follow-up studies.