The FDA has granted full approval to a new Alzheimer’s drug that has shown the ability to slow the progression of the devastating disease.
The approval of the drug, known as Leqembi, marks the first time that a drug meant to slow the progression of the disease has been granted full regulatory approval. Other approved drugs only target its symptoms.
“I don’t think we can understate the significance of this moment,” said Donna Wilcock, the assistant dean of biomedicine at the University of Kentucky.
About 6.7 million adults ages 65 and older in the United States have Alzheimer’s disease, according to the Alzheimer’s Association.
Leqembi, from Japanese drugmaker Eisai and U.S.-based drugmaker Biogen, targets a type of protein in the brain called beta-amyloid, long thought by scientists to be one of the underlying causes of Alzheimer’s disease.
In a phase 3 clinical trial of 1,795 patients with mild cognitive impairment or early-stage disease, the progression of the illness was slowed by 27% over an 18-month period.
“While patients still do decline on the drug, the decline is slowed,” Wilcock said.
Dr. Ronald Petersen, a neurologist at the Mayo Clinic in Rochester, Minnesota, said in an email that Leqembi is not a cure, nor does it stop the disease.
“It’s a first step for hopefully more therapeutics in the future,” he said.
The Alzheimer’s Association, which has vocally advocated for the drug’s approval, praised the decision.
The treatment could “give people in the early stages of Alzheimer’s more time to maintain their independence and do the things they love,” Joanne Pike, president, and CEO of the Alzheimer’s Association, said in a statement.
“This gives people more months of recognizing their spouse, children, and grandchildren,” Pike said.
“In real-world terms, this likely means more time for the patient to be living independently, enjoying their hobbies, their friends, and having a better quality of life,” Wilcock said. “Time will tell how much, but the clinical trial did show significant benefit on activities of daily living measures.”
Petersen said the drug appeared to slow a patient’s decline for about five months.
Who will be able to get Leqembi?
Leqembi was approved for people with mild cognitive impairment or early-stage Alzheimer’s disease.
The drug is given intravenously every two weeks, meaning patients will need to go to a hospital or clinic for the infusion.
In addition, a roundtable of Alzheimer’s experts recommends patients get periodic brain scans to monitor for any side effects.
Leqembi will carry a list price of $26,500 a year.
In June, the Centers for Medicare & Medicaid Services said it planned to provide coverage for Leqembi and other drugs in its class, contingent upon receiving full FDA approval. However, the agency said it will mandate that physicians gather real-world performance data on these medications through a government database. That means that only doctors who are willing to collect this data will be able to prescribe Leqembi.