US regulators have approved an Alzheimer’s drug from Biogen that split the scientific community, marking a significant moment for sufferers of the disease and their families.
Aducanumab is designed for patients suffering from mild to moderate Alzheimer’s. Biogen claims it reduces the progression of the disease by breaking up clumps of amyloid plaques that form in the brain.
The treatment has been surrounded by controversy for years, and many scientists opposed its approval, saying it does not work.
The US Food and Drug Administration’s decision announced on Monday is monumental given that no new drug has been approved for the disease in two decades, and very few treatments exist. Nearly 6m people in the US suffer from Alzheimer’s.
The regulator acknowledged the contentious nature of its decision.
“We are well-aware of the attention surrounding this approval,” said Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research.
She said that the treatment was “reasonably likely” to help patients but added that uncertainty remained about the drug’s clinical benefit. Ultimately the agency concluded that the overall benefits of aducanumab outweighed its risks for Alzheimer’s patients.
The FDA’s own panel of scientific advisers last year strongly opposed the approval of aducanumab, saying that Biogen’s trials did not prove sufficient evidence that the drug worked.
Cavazzoni acknowledged the disagreements but said the drug removed amyloid plaques from the brain and “it is expected” that this would reduce cognitive decline in people with the disease.
The drug’s approval was fast-tracked as it meets an unmet need for a severe disease and Biogen will need to carry out phase 4 trials.
The Alzheimer’s Association welcomed the decision, hailing it as a new day of hope for Alzheimer’s patients. “This approval is a victory for people living with Alzheimer’s and their families,” said Harry Johns, president of the Alzheimer’s Association.
John Hardy, professor of neuroscience at University College London, said: “While I am pleased that aducanumab has received approval, we have to be clear that, at best, this is a drug with marginal benefit which will help only very carefully selected patients.”
Trading in Biogen’s shares was halted ahead of the decision but shares in Japanese pharmaceutical company Eisai, which it partnered with, surged 45 per cent.
The FDA’s decision has ramifications for research into Alzheimer’s beyond Biogen. Shares in Eli Lilly, which is currently trialling its own amyloid-clearing drug, jumped 10 per cent.