The Food and Drug Administration approved Biogen’s Alzheimer’s drug aducanumab on Monday, making it the first U.S. regulator-approved drug to slow cognitive decline in people with Alzheimer’s and the first new drug for the disease in nearly two decades.
The FDA’s decision was eagerly awaited. The drug, which is marketed under the name Aduhelm, is also expected to generate billions in sales for the company offers new hope to friends and families of patients living with the disease. Biogen stock was on hold for the announcement.
“We are aware of the attention associated with this approval,” said Dr. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, in a press release. “We know that Aduhelm has drawn the attention of the press, the Alzheimer’s patient community, our elected officials and other interested stakeholders.”
“With treatment for a serious, life-threatening disease in balance, it makes sense that so many people followed the outcome of this review,” added Cavazzoni.
The FDA said it would continue to monitor the drug when it hits the US market. The agency granted approval on the condition that Biogen conduct another clinical study.
Alzheimer’s disease is a progressive neurodegenerative disease that slowly destroys memory and thinking skills. The Alzheimer’s Association estimates that more than 6 million Americans live with it. According to the group, this number is expected to rise to almost 13 million by 2050.
“It’s a new day,” said Harry Johns, CEO of the Alzheimer’s Association, in a statement. “This approval gives people with Alzheimer’s more time to live better. For families, it means being able to hold onto loved ones longer. It’s about resuscitating scientists and companies in the fight against this scourge of disease. It’s about hope it. “
To date, there have been no FDA-approved drugs that can slow the mental decline of Alzheimer’s, the sixth leading cause of death in the United States. The agency has approved Alzheimer’s drugs that are aimed at relieving symptoms rather than slowing the disease itself down.
Federal agencies have come under intense pressure from friends and family members of Alzheimer’s patients to speed up aducanumab, but the road to regulatory approval has been controversial since it showed promise in 2016.
In March 2019, Biogen stopped work on the drug after analysis by an independent group found it was unlikely to work. The company then shocked investors a few months later by announcing that it would apply for regulatory approval for the drug after all.
Biogen’s shares soared in November after the company received support from FDA officials who said the company had very “compelling” evidence of aducanumab’s effectiveness and “an acceptable safety profile that would make its use in individuals would assist with Alzheimer’s disease “, submitted.
But two days later, a panel of external experts advising the US agency unexpectedly declined to approve the experimental drug, citing inconclusive data. It also criticized the agency’s staff for rating it too positively.
When Biogen filed for approval for the drug in late 2019, its scientists said a new analysis of a larger data set showed that aducanumab “reduces clinical decline in patients with early-stage Alzheimer’s disease.”
Alzheimer’s experts and Wall Street analysts were immediately skeptical, wondering whether the clinical trial data was enough to prove the drug works and whether approval could make it difficult for other companies to enroll patients in their own drug trials.
Some doctors have said they won’t prescribe aducanumab when it hits the market because the mixed data package helps the company’s use.
Supporters, including advocacy groups and family members of patients desperately looking for a new treatment, have admitted the data is not perfect. However, they claim it could help some patients with Alzheimer’s, a progressive and debilitating disease.
Biogen’s drug targets a “sticky” compound in the brain known as beta-amyloid that scientists expect to play a role in the devastating disease. The company previously estimated that approximately 1.5 million people with early-stage Alzheimer’s in the United States could be candidates for the drug, according to Reuters.
The approval is “interesting because the FDA is essentially confirming that the beta-amyloid hypothesis has been validated,” said Salim Syed, a senior biotech analyst at Mizuho Securities, on Monday, adding that the decision had a major impact will have future clinical trials. Some experts are not convinced that targeting the compound will slow cognitive decline.
The FDA’s decision is expected to reverberate across the biopharmaceutical sector, RBC Capital Markets analyst Brian Abrahams said in a June 1 announcement to customers.
The US agency said Monday it found there was “substantial evidence” that the drug is helping patients. “With Aduhelm approved by the FDA, an important and critical new treatment is available to patients with Alzheimer’s disease to combat the disease,” the statement said.
