January 6, 2023 – The FDA has approved a brand new drug to treat early-stage Alzheimer's disease, despite the fact that clinical trial results showed only modest cognitive advantages.
The drug Lecanemab, brand name Leqembi, “is the latest therapy that targets the underlying disease means of Alzheimer's disease fairly than simply treating the symptoms of the disease,” said Billy Dunn, MD, director of the Office of Neuroscience in the FDA's Center for Drug Evaluation and Research, in a press release.
Like his controversial cousin Aducanumab Lecanemab was approved under the FDA’s accelerated approval process. put a drug on the fast track that offers a significant therapeutic advantage over existing treatments for a serious or life-threatening disease.
Modest benefits, side effects
The drug's label will state that “treatment with Leqembi must be initiated in patients with mild cognitive impairment or within the stage of mild dementia, the patient population during which the treatment was studied in clinical trials.”
The FDA approved the treatment based on clinical trials that found modest cognitive advantages in patients with early-stage Alzheimer's disease – but at the price of an increased risk of brain bleeding.
After 18 months of treatment within the clinical trial, lecanemab slowed cognitive decline by 27% in comparison with placebo.
The results are “Welcome news”, the difference is probably not clinically significant, A Current editorial within the Journal Thelancet CautionEd..
Also a newly published Case report in New England Journal of Medicine describes an Alzheimer's patient who died after taking Lecanemab.
Reaction from the Alzheimer's Association
Nevertheless, in anticipation of FDA's accelerated approval of lecanemab and the anti-amyloid drug donanemab, the Alzheimer's Association submitted a proper request to the Centers for Medicare & Medicaid Services last month for full and unrestricted reimbursement for FDA-approved Alzheimer's treatments.
“Every day counts when it comes to slowing the progression of this disease,” Joanne Pike, public health physician and president and CEO of the Alzheimer's Association, said in a press release on the time.
“Current CMS policy to severely restrict access to these treatments deprives people of options, leads to further, irreversible disease progression, and contributes to greater health disparities. This is unacceptable,” Pike added.
Following the announcement of today’s approval, Pike said in a brand new press release that the association “welcomes and celebrates this action by the FDA. We now have a second approved treatment that meaningfully changes the course of Alzheimer's disease for people in the early stages of the disease.”
Maria C. Carrillo, PhD, chief scientific officer of the Alzheimer's Association, called today's approval “a milestone.”
“The progress we have seen in recent years not only in this class of treatments but also in diversifying treatment types and targets is exciting and offers real hope to those affected by this devastating disease,” Carrillo said.
Critical issues
Alvaro Pascual-Leone, MD, PhD, professor of neurology at Harvard Medical School, said the FDA approval of lecanemab and its use in the clinic represents a “very exciting development and prospect; nevertheless, there are some critical issues that must be considered.”
In his opinion, the health system is “currently not prepared to meet the challenges and requirements of lecanemab” and future drugs.
“First, we need better workflows to identify appropriate patients who can benefit most from this treatment,” he says.