January 6, 2023 – The highly anticipated cure for Alzheimer’s disease was lecanemab granted expedited approval status to the FDA this afternoon, offering hope where there has been little for patients and their families affected by the devastating disease.
More than 6 million people in the US are living with Alzheimer’s disease.
It’s not a cure, but a drug given intravenously every 2 weeks has shown moderately positive effects in clinical practice attempts to slow down the early stage of the disease.
But many are cautious. As explained in an editorial last month in the magazine Lancet“The Alzheimer’s community has grown accustomed to false hope, disappointment and controversy.”
Some are concerned about the safety of lecanemab because some people in clinical trials experienced serious side effects of bleeding and swelling in the brain. Scientists recently attributed the third death to lecanemab, brand name Leqembi, although the drug manufacturer controversial the cause was medication.
So what should patients and their families make of today’s news? Here we answer some of the top questions related to the drug.
What does today’s FDA action mean?
The FDA granted accelerated approval to Leqembi after it showed positive trial results in slowing the progression of early-stage disease.
The FDA may grant expedited approval for drugs that treat serious conditions and meet unmet medical needs while drugs continue to be studied in larger studies.
With FDA approval in hand, doctors can now prescribe medicine.
Rebecca Edelmayer, PhD, senior director of scientific engagement for the Alzheimer’s Association, says that with today’s FDA move, speeding up production — and ultimately nationwide distribution and implementation — will take time.
“Ask your doctor about availability,” she says. “The main problem is that without insurance and Medicare coverage of this class of treatment, access for those who
could use the newly approved treatment, will be available only to those who can pay out of pocket. Without coverage, people simply don’t get treatment.”
The The Washington Post reports that with the expedited approval, drugmaker Eisai is expected to immediately seek full FDA approval, which would likely not come earlier this year. Full approval could help clear the way for Medicare coverage of the drug.
Those given Leqembi in an 18-month clinical trial experienced 27% less decline in memory and thinking compared to the placebo group. It also reduced brain amyloid, a sticky protein that builds up in the brains of people with Alzheimer’s 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 study in our field of a disease-modifying drug where clinical efficacy has been very clear.”
Concerns about side effects
The drug has raised safety concerns because it has been linked to some serious side effects, including brain swelling and bleeding. In the study, 14% of patients who received the drug experienced side effects which included brain swelling and bleeding, compared with about 11% in the placebo group.
Scientists have reportedly linked three death during a clinical trial with lecanemab, although it is unclear whether it caused death.
Fillit notes that the first two people who died were taking blood thinners when they received lecanemab.
“There are things about real-world drug use that we need to sort out, especially in the context of people with comorbidities,” he says.
The the third death is a little different, says Fillit. A patient who suffered a stroke showed signs of vasculitis, or inflammation of blood vessels.
“We don’t know exactly what happened, but we know it was very, very rare” among people involved in the trials, he says.
Edelmayer says the most common side effects reported during the trials were infusion-related reactions, headache and amyloid-related imaging abnormalities (ARIA). According to the FDA, these abnormalities “are known to occur with antibodies of this class. ARIA usually has no symptoms, although rarely serious and life-threatening events can occur.”
The FDA added them as a warning to the drug’s label, describing possible infusion-related reactions such as flu-like symptoms, nausea, vomiting and changes in blood pressure.
How much will it cost?
Eisai says lecanemab will cost $26,500 a year.
In a draft report released in December, the Institute for Clinical and Economic Evaluation (ICER) he listed a price ranging from $8,500 to $20,600 year would make the drug cost-effective. While the group has no power to set prices, many large health insurers consider its reports when negotiating prices, and some drug manufacturers take ICER recommendations into account when setting prices.
Editorial in Lancet warned last month that costs are likely to be “adverse” for low- and middle-income countries, and many health systems lack the infrastructure for widespread rollout.
Will Medicare cover it?
The Centers for Medicare and Medicaid Services (CMS), which runs Medicare, which covers most people with Alzheimer’s disease, said it will not widely cover amyloid-lowering drugs until the drug gets full approval in the US based on clinical benefits, unlike fast-track drugs . approval.
That means people would have to pay thousands out of pocket to get it first.
CMS decision it effectively denies Medicare coverage of FDA-approved fast-acting Alzheimer’s drugs unless the person is enrolled in an approved clinical trial.
On December 19, the Alzheimer’s Association filed a formal request asks CMS to remove the trial-only requirement and provide full and unlimited coverage for FDA-approved treatments for Alzheimer’s disease.
CMS says in declaration following today’s announcement: “Because Eisai’s product, lecanemab, has been granted accelerated FDA approval, it falls under CMS’s existing national coverage. CMS is reviewing the available information and may reevaluate its current coverage based on this review.”
“If lecanemab subsequently receives traditional FDA approval, CMS would provide broader coverage,” the statement said.
Who benefits the most from this medicine?
Lecanemab is a treatment for people with early stage Alzheimer’s who have amyloid in the brain. This means that people with other types of dementia or those in the later stages of Alzheimer’s disease are unlikely to get better with this drug.
Who makes Lecanemab?
The Japanese company Eisai is developing the drug, a monoclonal antibody, in collaboration with the American company Biogen.
What does the Alzheimer’s Association think?
The association urged expedited FDA approval. In a statementsays it “welcomes and is further encouraged” by the results of the clinical trials.
It is said that the data published in New England Journal of Medicine confirms that lecanemab “can significantly alter the course of the disease in people in the earliest stages of Alzheimer’s disease.”
“We are excited by the progress we are seeing in the research pipeline. The science tells us that while anti-amyloid treatment is not a cure – it will not be the end
treating Alzheimer’s — that’s certainly a start,” says Edelmayer.
Are there alternatives?
The FDA granted Biogen accelerated approval to make another Alzheimer’s drug, Aduhelm (aducanemab), in 2021, but the move was controversial because the drug’s effectiveness was widely questioned. It has since been largely withdrawn from the market.
Aduhelm was the first approved treatment for early-stage Alzheimer’s disease since 2003.