FDA grants full approval to Leqembi, first drug to slow progress of Alzheimer’s
Ren Willie likes to tell simple jokes and sprinkle puns into conversations. Those jokes and puns, he says, are always clean and basically on a junior high level. That makes sense because he was a junior high resource and health teacher for many years.
The jokes are vivid, tucked safely in his long-term memory to be pulled out whenever he wants to share them.
His short-term memory is more hit-and-miss, as if occurrences and thoughts were tossed somewhat haphazardly in random drawers in a slightly messy house. Some are easy to find and others have been misplaced, though he may stumble onto them.
A year ago, Willie, 80, who lives in Murray, Utah, was diagnosed with Alzheimer’s dementia, a progressive neurodegenerative disease that over time destroys mental functions including memory. His doctor put him on a couple of different medications that seem to help symptoms and mood, but won’t slow the disease.
So he and his wife of 45 years, Jane Willie, were among Alzheimer’s patients and their families paying close attention Thursday when the U.S. Food and Drug Administration announced full approval of lecanemab for early-stage Alzheimer’s. It’s the first drug shown to delay the progression of the disease.
“Today’s action is the first verification that a drug targeting the underlying disease process of Alzheimer’s disease has shown clinical benefit in this devastating illness,” said Teresa Baracchio, acting director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, in a written statement. “This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease.”
The approval came with a warning on the drug’s label, noting that in rare cases there have been “serious and life-threatening events.”
The national Alzheimer’s Association heralded the decision in a press release.
“This treatment, while not a cure, can give people in the early stages of Alzheimer’s more time to maintain their independence and do the things they love,” said Joanne Pike, Alzheimer’s Association president and CEO. “This gives people more months of recognizing their spouse, children and grandchildren. This also means more time for a person to drive safely, accurately and promptly take care of family finances, and participate fully in hobbies and interests.”
The treatment from drug makers Eisai and Biogen, marketed as Leqembi, already has accelerated approval. But the Centers for Medicare and Medicaid Services (CMS) said without full approval it would not pay for the drug for Medicare beneficiaries outside of clinical trials — a decision that sparked a firestorm. Thursday’s decision by the FDA means that the drug will be at least somewhat more accessible to seniors struggling with early Alzheimer’s.
It’s not clear that Ren will qualify and his doctor may not ever prescribe it, the Willies told Deseret News. Or that he would choose to take the drug, which has the potential for significant side effects. But they feel strongly that medications approved by the FDA should be available, the decision between doctor, patient and pocketbook. Those with Alzheimer’s typically look to Medicare for help with costs.
CMS decision ignites sparks
Medicare coverage for monoclonal antibodies for Alzheimer’s has been contentious. Deseret News first met Ren Willie at a protest held by the Alzheimer’s Association of Utah at the state Capitol in late June. It was one of many protests organized nationally to bring attention to what the association called the “unprecedented” decision by CMS to deny coverage for lecanemab and other monoclonal antibody therapies for Alzheimer’s.
The denial came for an earlier drug, aducanumab (marketed as Aduhelm) but CMS officials said it would also apply to future monoclonal antibody treatments approved on an accelerated basis against Alzheimer’s. They never clearly explained the reason for the denial, though speculation was the degree of efficacy and cost of aducanumab. And critics of the drug said the approval process had been problematic. But CMS said the decision would apply to future Alzheimer’s disease drugs in the same class, too.
Monoclonal antibodies are lab-created proteins. In the case of the Alzheimer’s drugs, treatments are given as infusions that bind to and clear beta amyloid plaques in the brain that are considered a significant factor in development of Alzheimer’s. Clearing them reduces tau protein tangles that interfere with brain function. The plaques can be seen on positron emission tomography scans years before symptoms appear. It’s believed clearing the plaque slows the disease, though it doesn’t stop it or reverse damage.
Lecanemab is only approved for early Alzheimer’s and there have been some side effects reported, including rare but concerning brain bleeds. The drug slows cognitive impairment by about 27%, according to the clinical trials.
As Deseret News earlier reported, when CMS said it would not cover the medication, backlash was fierce, including in congressional committee meetings and protests like the one at the Utah Capitol. In letters to Health and Human Services Secretary Xavier Becerra and CMS administrator Chiquita Brooks-LaSure, 74 U.S. House members and 20 senators asked for reconsideration. Brooks-LaSure finally said if the drugs received full FDA approval, CMS would cover them.
Groups like the Alzheimer’s Association note time is of the essence. Because lecanemab is only approved for those with mild cognitive impairment and early stage Alzheimer’s, the treatment window is narrow.
Meanwhile, the Veterans Administration said it would provide the medication to eligible veterans over 65. “That raises the specter of a veteran being treated while a spouse in a similar stage of Alzheimer’s could be denied the treatment,” Deseret News reported.
Who will get it and how?
This week’s approval doesn’t mean the drug will be easy to get or to afford. Access for Medicare beneficiaries could be limited because Brooks-LaSure said prescribing doctors must participate in registries that compile data. There are questions about who will run the registries and whether doctors who might otherwise prescribe the drug could deem that too much extra work, Jeremy Cunningham, of the Utah Alzheimer’s Association chapter, told Deseret News.
The cost is high. The drugs are given in twice monthly infusions, the cost set at $26,500 a year. That means that even with Medicare coverage for those with the drug benefit, the out-of-pocket cost can be thousands. Patients receiving it also need MRIs at certain points to look for brain swelling and bleeds. And to qualify, they’ll need tests to verify amyloid accumulation. Insurance doesn’t cover those typically. They may also need gene testing, because certain genes make complications more likely.
Related
KFF, formerly the Kaiser Family Foundation, said the cost for Medicare could lead to rate increases for all seniors enrolled in the program. It estimates that if 10% of the close to 7 million older adults with Alzheimer’s take the drug, spending would exceed that of the top 10 Part B drugs administered in doctors’ offices in 2021.
But the Willies believe access to the drug was worth rallying around. Once a drug is approved, even in accelerated form, it should be covered, they said.
Reason to hope
Neuroscience News recently called lecanemab “promising,” which is a positive adjective in a research field that’s had more bad news than good for many years.
Experts say drugs like lecanemab that offer some improvement and reason to hope are a good start in the search for treatment, prevention or cure.
In the meantime, the Willies have a simple test that hints at what kind of a day Ren will have. Jane asks him how he’s doing and he gives her a thumbs up or a thumbs down. Sometimes, the thumb goes sideways to say so-so. He’s accepted his diagnosis now, he says, though he went through denial and grief and still gets frustrated.
On good days, he volunteers at a local senior center, helping teach older adults to play the harmonica. He started playing it when he was in the Navy. He was a missile technician on a nuclear sub and says facetiously that fellow sailors sent him to the bilge and told him not to come back up until he played better.
This week, his harmonica version of “Amazing Grace” was flawless.
Ren’s writing what he calls “bits of my history before I forget.” He wants to pass his precious memories down to his four children and grandchildren.
“He has a good attitude,” says Jane. “It’s a challenge, but we’re making lemons into lemonade as much as we can.”
