FDA fully approves new Alzheimer’s drug to slow down disease progression

‘Today’s action is first verification that a drug targeting underlying disease process has shown clinical benefit’

The US Food and Drug Administration has fully approved a new drug meant to slow down the progression of Alzheimer’s disease, allowing for the medicine’s wider insurance coverage.

Estimates suggest that more than 6.5 million adults of ages 65 and over in the US live with Alzheimer’s disease, which can slowly destroy people’s memory and thinking skills and eventually, their ability to carry out even simple everyday tasks.

The FDA’s full approval for Leqembi marks a new milestone in treating the condition as the drug is the first approved therapeutic meant to slow down the progression of the disease, compared to others that work by targeting symptoms.

Leqembi works by reducing the levels of amyloid plaques in the brain, a hallmark feature of Alzheimer’s disease.

While the specific causes of Alzheimer’s are not fully known, the neurological condition is characterised by changes in the brain, such as the formation of amyloid beta plaques, that have been linked to the loss of nerve cells and their connections.

Prior to the latest approval, the FDA required the applicant to conduct a clinical trial to verify the anticipated clinical benefit of the drug.

Researchers conducted a multicenter, randomised clinical trial, enrolling 1,795 patients with Alzheimer’s disease, including those with mild dementia stage of the disease, to verify the drug’s effectiveness.

Patients were randomly assigned in a 1:1 ratio to either receive a placebo or a calculated dose of Leqembi once every two weeks.

Scientists found a “statistically significant and clinically meaningful reduction” of cognitive decline in the patients taking the drug compared to those in the placebo group.

However, researchers also found some side-effects of Leqembi, including headache, as well as amyloid-related imaging abnormalities (ARIA) – a side effect known to occur with the class of antibodies targeting the amyloid plaques.

The latter condition, they explained, presents as temporary swelling in areas of the brain that usually resolves over time and may be accompanied by small spots of bleeding in or on the surface of the brain.

ARIA, according to scientists, can occur with symptoms like headache, confusion, dizziness, vision changes, and nausea, and may also present with “serious and life-threatening brain edema,” linked to “seizures and other severe neurological symptoms,” the FDA noted.

Considering these potential effects of the drug in some patients, a boxed warning is to be included in the prescribing information to alert patients and caregivers to the potential risks associated with ARIA, the federal drug regulator said.

Leqembi is also to be initiated in patients with mild cognitive impairment or mild dementia stage of Alzheimer’s disease – the group on whom the drug was tested in the clinical trial.

“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 disease,” Teresa Buracchio, acting director of the Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research said in a statement.

“This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer’s disease,” Ms Buracchio said.

The US federal agency Centers for Medicare & Medicaid Services announced following the FDA approval that broader insurance coverage is now available for the drug.

“CMS today affirms our commitment to help people with Alzheimer’s disease have timely access to innovative treatments that may lead to improved care and better outcomes,” CMS Administrator Chiquita Brooks-LaSure said.

“With FDA’s decision, CMS will cover this medication broadly while continuing to gather data that will help us understand how the drug works. This is welcome news for the millions of people in this country and their families who are affected by this debilitating disease,” Ms Brooks-LaSure added.