Controversial Alzheimer’s drug becomes first new treatment approved in US in almost 20 years

The US Food and Drug Administration on Monday approved the use of Aducanumab to treat Alzheimer’s, the first medication of its kind approved in nearly 20 years. An outside panel of experts cautioned the FDA against approving the drug last year, warning that it wasn’t effective enough for treatment. The drug will be marketed under the name Aduhelm.

“Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones,” said Dr Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research.

6.2 million Americans are affected by the disease. The FDA said the drug was approved using an accelerated approval pathway, a process used when a drug for a serious or life-threatening illness “provides a meaningful therapeutic advantage over existing treatments”.

“Currently available therapies only treat symptoms of the disease,” Dr Cavazzoni said. “This treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s. As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.”

The drug is administered as a monthly intravenous infusion to slow down cognitive decline in patients who are in the early stages of the disease.

Experts on the Peripheral and Central Nervous System Drugs Advisory Committee recommended to the FDA in November that the drug be rejected following conflicting results in two clinical trials.

According to a document released in the fall, FDA reviewers found “substantial evidence of effectiveness”. But the same document also stated that a reviewer had raised questions concerning statistical methods, adding that they had not been convinced by the evidence and recommended another trial of the drug.

Alzheimer’s is a progressive and irreversible disease that destroys a person’s memory and thinking abilities. Eventually, just performing simple tasks becomes difficult.

Aduhelm is the first drug approved for the treatment of Alzheimer’s since 2003. The studies performed to test the effectiveness of the drug included a total of 3,482 patients.

The prescribing information for the drug includes a warning for “amyloid-related imaging abnormalities,” which most often presents itself as “temporary swelling in areas of the brain,” according to the FDA.

“Some people may have symptoms such as headache, confusion, dizziness, vision changes, or nausea,” the agency said. They added that the most common side effects of Aduhelm were amyloid-related imaging abnormalities, headache, fall, diarrhoea, and confusion or disorientation.  

The FDA said they were requiring the maker of the drug, Biogen, to “conduct a new randomized, controlled clinical trial to verify the drug’s clinical benefit”.

“If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug,” the agency said.

The CEO of the company based in Cambridge, Massachusetts, Michel Vounatsos said the approval of the drug comes after more than a decade of research.

“We believe this first-in-class medicine will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come,” he said in a statement. “We are grateful for the contributions of thousands of patients and caregivers who participated in our clinical trials, as well as for the dedication of our scientists and researchers.”