January 6, 2023 – The highly anticipated Alzheimer’s drug Lecanemab was Accelerated approval grantedThe status released by the FDA this afternoon offers hope to patients and their families affected by this devastating disease where there has previously been little.
More than 6 million people within the United States live with Alzheimer's disease.
It shouldn't be a cure, however the drug, which is run intravenously every two weeks, has shown moderate positive effects in the clinic Attempts to decelerate the early stages of the disease.
But many are suspicious. As an editorial in last month's magazine The Lancet“The Alzheimer’s community has become accustomed to false hope, disappointment and controversy.”
Some are concerned concerning the safety of lecanemab because some patients in clinical trials experienced severe unintended effects akin to bleeding and swelling within the brain. Scientists recently a third death attributed to Lecanemab, brand name Leqembi, although the drug manufacturer controversial The medication was the cause.
So what should patients and their families make of today's news? Here we answer a few of an important questions surrounding the drug.
What does today’s FDA motion mean?
The FDA granted Leqembi accelerated approval after studies showed positive leads to slowing the progression of early-stage disease.
The FDA can accelerated approval for medicines to treat serious diseases and to handle unmet medical needs while the medicines proceed to be studied in larger trials.
With FDA approval in hand, doctors can can now prescribe the medication.
Dr. Rebecca Edelmayer, senior director of scientific engagement on the Alzheimer's Association, said today's FDA move will take time to ramp up production and eventually enable nationwide distribution and rollout.
“Ask your doctor about availability,” she says. “The main problem is that without insurance and Medicare coverage of this class of treatment, access is limited for those who
“The treatment that could benefit those who could benefit from the newly approved treatment will only be available to those who are able to pay for it out of pocket. Without health insurance, people simply won't be able to get the treatment.”
The Washington Post reports that with the accelerated approval, drugmaker Eisai is expected to immediately seek full FDA approval, which likely won't happen until later this year. Full approval could help clear the way for Medicare to cover the drug.
Possible benefits?
Those who received Leqembi for 18 months in a clinical trial experienced a 27 percent smaller decline in memory and thinking skills than the group that received a placebo. It also reduced brain amyloid, the sticky protein that builds up in the brains of Alzheimer's patients and is considered a hallmark of the disease.
Howard Fillit, MD, co-founder and chief scientific officer of the Alzheimer's Drug Discovery Foundation, says, “This is the first Phase III trial of a disease-modifying drug in our field that has shown very clear clinical efficacy.”
Concerns about side effects
The drug has raised safety concerns because it is associated with certain serious side effects, including brain swelling and bleeding. In the study, 14% of patients who received the drug showed Side effects These included brain swelling and bleeding, compared with about 11% in the placebo group.
Scientists have reportedly discovered three Deaths during the clinical trial of lecanemab, although it is unclear whether it caused the deaths.
Fillit points out that the first two people who died had taken blood thinners when Lecanemab was administered.
“There are things about real-world use of the drug that we need to clarify, particularly in the context of people with comorbidities,” he says.
The third death is a little different, says Fillit. The patient, who had suffered a stroke, showed signs of vasculitis, i.e. inflammation of the blood vessels.
“We don't know exactly what happened, but we know that it was very, very rare among the people involved in the trials,” he says.
According to Edelmayer, the most commonly reported side effects during the trials were infusion-related reactions, headaches and amyloid-related imaging abnormalities (ARIA). According to the FDA, these abnormalities are “known in antibodies of this class. ARIA usually does not result in symptoms, although in rare cases it can cause serious and life-threatening events.”
The FDA added these warnings to the drug's package insert, describing the possible infusion-related reactions as flu-like symptoms, nausea, vomiting, and changes in blood pressure.
How much will it cost?
Eisai says Lecanemab will cost $26,500 per year.
In a draft report published in December, the Institute for Clinical and Economic Review (ICER) writes said a price between $8,500 and $20,600 a year would make the drug cost-effective. Although the group has no power to set prices, many large health insurers consider its reports when negotiating prices, and some drug manufacturers consider ICER recommendations when setting prices.
An editorial in The Lancet warned last month that the cost would likely be “prohibitive” for low- and middle-income countries and that many health systems didn't have the infrastructure for widespread rollout.
Does Medicare cover the prices?
The Centers for Medicare and Medicaid Services (CMS), which runs the federal government's medical health insurance program for many Alzheimer's patients, has indicated that it can not widely cover amyloid-lowering drugs until the drug receives full U.S. approval (reasonably than accelerated approval) based on its clinical profit.
This means that folks would initially should pay 1000's out of their very own pocket to receive this service.
The CMS decision Medicare effectively refuses to cover fast-track, FDA-approved Alzheimer's drugs unless the person participates in an approved clinical trial.
On December 19, the Alzheimer's Association filed a formal request We ask CMS to remove the trial-only requirement and supply full and unrestricted coverage for FDA-approved Alzheimer's treatments.
CMS says in a statement following today's announcement: “Because Eisai's product lecanemab received accelerated approval from the FDA, it falls under CMS' existing national coverage determination. CMS is reviewing the available information and may reconsider its current coverage based on that review.”
“If lecanemab subsequently receives traditional FDA approval, CMS would provide broader coverage,” the statement said.
Who advantages most from this medicine?
Lecanemab is a drugs for individuals with early-stage Alzheimer's disease who've amyloid of their brain. This means that folks with other forms of dementia or people in late-stage Alzheimer's disease are unlikely to enhance with this medicine.
Who makes Lecanemab?
Japan-based Eisai is developing the drug, a monoclonal antibody, in collaboration with US company Biogen.
What is the Alzheimer's Association's view?
The association pushed for accelerated approval by the FDA. In a statementThe company says it “welcomes and continues to be encouraged” by the clinical trial results.
It is claimed that data published within the New England Journal of Medicine confirms that lecanemab “can significantly alter the course of disease in people with early-stage Alzheimer’s disease.”
“We are excited about the progress we are seeing in the research pipeline. Science tells us that while anti-amyloid treatments are not a cure, they are not the end of
Treatment of Alzheimer's disease – they are certainly the beginning,” says Edelmayer.
Are there alternatives?
The FDA granted Biogen accelerated approval in 2021 to supply one other Alzheimer's drug, Aduhelm (aducanemab). However, the move was controversial because the drug's effectiveness was widely questioned, and it has since been largely withdrawn from the market.
Aduhelm was the primary approved treatment for early-stage Alzheimer's disease since 2003.