Hope for Alzheimer’s patients: a new treatment against the disease has just been approved on June 7 by the American authorities for the first time in two decades.
Named Aduhelm and produced by the pharmaceutical company Biogen, it is the “first treatment approved for Alzheimer’s disease since 2003,” said the US Agency for Drugs (FDA) in a statement.
The cost of the product will drop to about $ 56,000 per year for a middleweight American, the company said on June 7. Its reimbursement will depend on the health coverage to which each patient has subscribed.
Read also: Alzheimer’s: hope for a new avenue of prevention?
Slow down the disease
It is the first treatment directed against the disease mechanism itself, “the presence of beta-amyloid plaques in the brain,” and not just the symptoms it causes, the company explains.
“We are delighted with this historic decision”, reacted the American organization Alzheimer Association. “It is the first drug approved by the FDA to slow the decline due to the disease,” President Harry Johns said in a statement.
How does this treatment work?
The drug uses a molecule, called aducanumab. These are monoclonal antibodies that aim to dissolve aggregates of a protein, beta-amyloid, the accumulation of which creates plaques in the brain tissue of patients. Which leads to Alzheimer’s pathology.
This target is one of the main avenues explored by research against this degenerative pathology.
But treatment studies have suffered setbacks in recent years, and no cure exists to date. This is why this drug, administered intravenously every four weeks, was seen as a silver lining.
Still need other medication
In accordance with the great debate that agitates the scientific community around this treatment, some experts have tempered the enthusiasm on June 7.
“While I am happy that aducanumab has received clearance, we need to be clear that at best this drug will have marginal benefit that will only help some carefully chosen patients,” said John Hardy, professor of neuroscience. at University College London. “We will need better medicine in the future.”
For the scientific director of the Alzheimer Association, this is precisely what the authorization of the FDA will allow to aim: “History has shown that an authorization of a first drug of a new category stimulated the sector. , increased investments in new treatments and encouraged innovation, “said Maria Carrillo, quoted in the organization’s statement.
Efficiency in question
In November, however, an expert committee ruled against authorizing this treatment, judging that it had not sufficiently demonstrated its effectiveness. A non-binding opinion, but it is rare for the FDA to dispense with it.
The committee had studied data from two studies: one large clinical trial concluded that the drug from Biogen laboratories was effective, while another had given negative results.
But “the FDA has determined that there is substantial evidence that Aduhelm reduces beta-amyloid plaques in the brain and that reducing these plaques is reasonably likely to result in significant benefits for patients,” a- she declared. The Agency thus “concluded that the benefits (…) outweigh the risks” of taking this drug, she added.
A conditional authorization
The FDA has also used a fast-track procedure that only grants the product conditional authorization. This possibility had not been studied by the committee.
“The FDA asks Biogen to conduct post-authorization clinical trials to verify the clinical benefits of the drug,” said the US regulator. “If the drug does not work as expected, we may take steps to take it off the market.”
An “urgent” need
Almost 6 million Americans live with Alzheimer’s, which is the sixth leading cause of death in the United States. The disease gradually robs the memory of affected patients, who in the later stages of the disease can no longer perform daily tasks or hold conversations.
“The need for treatment is urgent,” hammered the FDA. “I think we have clearly heard from patients that they are ready to accept a certain uncertainty in order to have access to a drug that could produce significant effects”, argued at a press briefing Peter Stein, head of within the Agency.