US officials have granted full approval to the closely watched Alzheimer’s disease drug Leqembi.
Key points:
- Leqembi is the first medicine that has been convincingly shown to modestly slow the cognitive decline caused by Alzheimer’s disease
- Early test results suggested Leqembi worked by clearing a sticky brain plaque linked to the disease
- The FDA confirmed those results by reviewing data from a larger, 1,800-patient study
The US Food and Drug Administration (FDA) endorsed the IV drug for patients with mild dementia and other symptoms caused by early Alzheimer’s disease.
It is the first medicine that has been convincingly shown to modestly slow the cognitive decline caused by Alzheimer’s.
On June 30 Australia’s Therapeutic Goods Administration (TGA) accepted and commenced evaluating an application to register the use of Leqembi, also known as lecanemab, in Australia.
The legislated time frame for TGA assessment is 255 working days for applications of this kind but time frames for evaluation of individual applications ultimately depend on when all data is provided by the sponsor.
The TGA’s assessment will consider the risks and benefits of the treatment and will ensure that the data meets its standards of safety, quality and efficacy.
Japanese drug maker Eisai received conditional approval from the FDA in January based on early results suggesting Leqembi worked by clearing a sticky brain plaque linked to the disease.
The FDA has now confirmed those results by reviewing data from a larger, 1,800-patient study in which the drug slowed memory and thinking decline by about five months in those who got the treatment, compared to those who got a dummy drug.
“This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease,” The FDA’s neurology drug director, Teresa Buracchio, said.
The drug’s prescribing information will carry the most serious type of warning, indicating that Leqembi can cause brain swelling and bleeding, side effects that can be dangerous in rare cases.
The label notes that those problems are seen with other plaque-targeting Alzheimer’s drugs.
The process of obtaining full FDA approval for a drug usually attracts little attention. But Alzheimer’s patients and advocates have been lobbying the federal government for months after US Medicare officials announced last year they would not pay for routine use of drugs such as Leqembi until they received the FDA’s full approval.
There were concerns that the cost of new, plaque-targeting Alzheimer’s drugs could overwhelm the finances of the program, which provides care for 60 million seniors. Leqembi is priced at about $US26,500 ($39,974) for a year’s supply of IVs every two weeks.
The vast majority of Americans with Alzheimer’s get their health coverage through Medicare. And private insurers have followed its lead by withholding coverage for Leqembi and a similar drug, Aduhelm, until they receive FDA’s full endorsement.
An FDA decision on full approval for Aduhelm is still years away.
Medicare administrator Chiquita Brooks-LaSure said the program would begin paying for Leqembi now that it had full FDA approval.
But the US government is also setting extra requirements, including enrolment in a federal registry to track the drug’s real-world safety and effectiveness.
“[Medicare] will cover this medication broadly while continuing to gather data that will help us understand how the drug works,” the administrator said.
Some Medicare patients could be responsible for paying the standard 20 per cent of the cost of Leqembi, though the amount will vary depending on their plans and other coverage details.
Hospitals and medical clinics have cautioned that it may take time to get people started on the drug.
Doctors need to confirm that patients have the brain plaque targeted by Leqembi before prescribing it.
Nurses need to be trained to administer the drug and patients must be monitored with repeated brain scans to check for swelling or bleeding.
The imaging and administration services carry extra costs for hospitals beyond the drug itself.
Eisai has told investors that about 100,000 Americans could be diagnosed and eligible to receive Leqembi by 2026.
“We want to ensure that appropriate patients only are the ones that get this product,” Alexander Scott, a vice president with Eisai, said.
Eisai studied the drug in people with early or mild disease who were evaluated using a scale measuring memory, thinking and other basic skills.
After 18 months, those who got Leqembi declined more slowly — a difference of less than half a point on the scale — than participants who received a dummy infusion.
Some Alzheimer’s experts say that delay is likely too subtle for patients or their families to notice.
But federal health advisers said the difference could still be meaningful and recommended that the FDA fully approve the drug at a public meeting in June.
AP