Alzheimer's is the 6th leading cause of death in the US
US regulators on Monday approved Biogen Inc's aducanumab as the first treatment to attack a likely cause of Alzheimer's disease despite controversy over whether the clinical evidence proves the drug works.
Aducanumab aims to remove sticky deposits of a protein called amyloid beta from the brains of patients in earlier stages of Alzheimer's in order to stave off its ravages, which include memory loss and the inability to care for one's self.
"This is good news for patients with Alzheimer's disease. We've not had a disease-modifying therapy approved ever," said Dr Ronald Petersen, an Alzheimer's disease expert at the Mayo Clinic. However, he cautioned, "This is not a cure. It's hoped that this will slow the progression of the disease."
"I think this is a big day," Dr Peterson said. "But we can't overpromise."
Alzheimer's is the sixth-leading cause of death in the US.
The Food and Drug Administration (FDA) said on its website that clinical trials for the treatment, to be sold under the brand name Aduhelm, showed a reduction in the plaques that is expected to lead to a slower decline in patients.
"Although the Aduhelm data are complicated with respect to its clinical benefits, FDA has determined that there is substantial evidence that Aduhelm reduces amyloid beta plaques in the brain and that the reduction in these plaques is reasonably likely to predict important benefits to patients," the agency said in a statement.
Biogen shares rose as much as 3% on Monday before being halted up 0.1% at $286.42 ahead of the FDA decision. The US traded shares partner Eisai Co rose $33.50, or 45%, to $107.75
The price of the treatment was not immediately available.
Biogen's drug had been hailed by patient advocates and some neurologists eager to have an effective option for patients with the lethal disease. Other doctors said clinical trial results were inconsistent and more proof was needed.
'A win-win for all'
Current Alzheimer's treatments including AbbVie's Namenda and Pfizer's Aricept are intended to ease Alzheimer's symptoms, while the Biogen drug is the first-ever approved to target the underlying cause of dementia and slow progression.
The FDA said Biogen will need to conduct a post-approval trial to verify Aduhelm's clinical benefit, and that it could be pulled from the market if the drug does not work as intended.
"I think they made the right decision. It makes the drug available to patients while requiring the company to do more research to prove its benefit. I think it's a win-win for all," said Dr. Howard Fillit, chief science officer of the Alzheimer’s Drug Discovery Foundation.
Aducanumab was studied in patients with early disease who test positive for a component of amyloid brain plaques.
Some trial patients in trials experienced potentially dangerous brain swelling. The FDA advised that patients who experience the side effect should be monitored but not necessarily taken off the drug.
The FDA decision did not define the specific Alzheimer's patients for who the treatment is appropriate – such as very early stage disease - raising the possibility that the patient population could be larger than anticipated. The FDA was not immediately available for comment.
Biogen has estimated that around 1.5 million Americans would be eligible for treatment with aducanumab, which is given by monthly infusion, raising concerns about costs to the healthcare system.
The number of Americans living with Alzheimer’s is expected to rise to around 13 million by 2050 from more than six million currently, according to the Alzheimer’s Association.
"We think the drug has the potential to generate (at least) $10 billion in peak sales, and believe a successful launch of aducanumab has the potential to completely change the profile of the company," said Guggenheim analyst Yatin Suneja.
Biogen and partner Eisai Co had scrapped development of the drug in March 2019, then reversed course in October, saying a more detailed data analysis showed some patients with very early forms of the disease benefited from taking higher doses over an extended period of time.
Wall Street analysts had forecast that an FDA approval of Biogen's drug could reinvigorate a field that has been abandoned by many large pharmaceutical companies.
The amyloid theory has been at the center of Alzheimer's research for many years with a long list of failures to show for it, and seemed on life support until Biogen revived its aducanumab program.
Shares of companies developing Alzheimer's treatments also jumped, with AC Immune SA and Anavex Life Sciences up between 14% and 17%, while shares of Eli Lilly and Co and Axsome Therapeutics rose more than 5%.
The Institute for Clinical and Economic Review, an influential drug pricing research group, in a May report cited "insufficient" evidence that aducanumab provides a net health benefit. It said data so far indicate a cost-effective price of no more than $8,300 per year.
Looking only at favorable trial results, that price rises as high as $23,100, ICER said
The research group put the fair price for any drug proven to halt the progression of Alzheimer’s-related dementia at $50,000 to $70,000 per year.