The pharmaceutical industry is looking forward to the U.S. regulator’s decision on a potential blockbuster drug Alzheimer’s. The drug is controversial but, if approved, would be the first new treatment for debilitating illness in years.
The U.S. Food and Drug Administration is scheduled to announce on Monday whether or not to approve the drug Biogen addaninum, which aims to slow the progression of Alzheimer’s disease.
Why is this important?
If aducanumab is approved, it would be one of the most significant decisions the FDA has made in years. For many Alzheimer’s patients and their families, it would be hailed as revolutionary in a field where there are few treatments and no new drugs they have been approved since 2003.
But the drug, which Biogen has co-authored with Japanese company Eisai, has sparked controversy, with many saying the treatment does not work alone.
In January, the FDA reversed its decision after months of delays. Pharmaceutical investors, as well as 6m people living with Alzheimer’s in the United States, are nervously awaiting the regulator’s conclusion.
“Without a doubt this is the biggest binary event in biopharma in 2021,” said Colin Bristow, UBS analyst, referring to the drug’s approval or rejection.
How does the drug work?
Aducanumab is a monoclonal antibody treatment that is administered intravenously to patients with moderate Alzheimer’s disease.
Biogen says the drug binds to beta amyloid molecules that create plaques in the brain and break down cells, thus reducing the progression of Alzheimer’s so people can continue to do daily activities such as cleaning and shopping.
The drug test u ”amyloid hypothesis”, Who argue that they believe that plaque accumulation in the brain causes Alzheimer’s.
Howard Fillit, founding executive director of the Alzheimer’s Drug Discovery Foundation, called it “the first rigorous test of the amyloid hypothesis.”
What are the controversies surrounding the drug?
Many scientists dispute that aducanumab works at all after a series of problems with Biogen’s clinical trials.
In March 2019, Biogen signed his two lawsuits after an independent committee said the drug was not going to be effective. But in October 2020, the company said a new analysis of a larger set of test data showed that the drug worked when given at a higher dose.
Biogen has also been criticized for providing analysis after its tests were concluded and some scientists say its data are insufficient to deserve regulatory approval. In November, an FDA advisory panel ripicca to the agency’s results of “Substantial evidence” that aducanumab worked.
“There’s a clear and well-defined bar that a product needs to be able to overcome to access the market and that product doesn’t,” said Caleb Alexander, a professor at the Johns Hopkins Bloomberg School of Public Health, and one of the panel members.
“I will step out of my chair if they approve it,” he said, adding that a green light “would mark one of the most consistent and poor decisions the FDA has made in recent history.”
Why do so many people oppose the drug?
A chain of pharmaceutical companies have tried and failed to test the amyloid hypothesis and create a drug that slows the progression of Alzheimer’s. Little evidence exists that amyloid cleansing reduces disease progression.
Aborted lawsuits include Pfizer and the Johnson & Johnson discarded study in 2012 and Merck’s failed lawsuit in 2017. Last year, Eli Lilly and Roche’s two drugs both also slowed the rate of cognitive decline.
What happens if it is approved?
Approval will be hailed by those living with Alzheimer’s as a way forward, as well as a claim by Biogen who has fought criticism of his management of drug development for years.
The Alzheimer’s Association has ordered the FDA to approve aducanumab, saying any treatment that gives patients more time is worthwhile.
Analysts expect the drug to become a successful seller for Biogen, although revenue estimates vary.
Matthew Harrison, head of biotechnology research at Morgan Stanley, said the demand for patients is likely to be vast, but some doctors have refused to administer addannumab even if they receive approval. “Investors aren’t sure they understand what that dynamic will ultimately mean.”
It expects Biogen to garner $ 7 billion in sales by 2030 while UBS’s Bristow expects maximum annual sales of $ 18 billion worldwide.
“It would be one of the biggest drugs in the industry,” Bristow said.
What happens if it is rejected?
Aducanumab rejection will be a blow to people suffering from Alzheimer’s who have raised their hopes for a slower mental decline from the drug.
More than 6 million people in the United States live with the disease, according to the Alzheimer’s Association, and many conditions are worse during the coronavirus pandemic that contact between their lovers has been reduced to slow the spread of the virus.
The refusal will also be a heavy blow to Biogen, which has spent millions on drug development and has fallen into other areas. The company’s shares have fluctuated widely since previous announcements about the drug.
“Aducanumab has really hurt Biogen for a while because it was such an overwhelming focus of the company, they are back in gene therapy. [and] new neurodegenerative treatments, ”said Tom Shrader, general manager of BTIG.
What does the FDA’s decision mean for other companies?
A green light from the FDA will raise confidence in the amyloid hypothesis and increase investment in further studies. Eli Lilly is trying donanemab, another drug that eliminates amyloid, and positive news for Biogen should be able to push sentiment toward Lilly’s study.
Geoff Meacham, an analyst at Bank of America, said a rejection “will not necessarily put a nail in the coffin of the amyloid hypothesis” because Biogen’s controversial processes are difficult to compare with others. Meanwhile, imaging technologies have progressed so that patients can be more effectively examined to see if they have amyloid in their brain in the first place.