On Monday, a 17-year drought in the world of Alzheimer’s drugs ended with the FDA approval of Biogen’s Eduhalem (edukanumab). The controversy behind the FDA’s decision was considerable, but it doesn’t seem to scare drug developers who are now narrowing down on degenerative brain disease.
In short, Aduhelm’s approval was obtained after conflicting results from clinical trials. In November 2020 An independent FDA advisory board did not recommend that the agency endorse the drug, but in June, the agency approved the drug through an accelerated approval program anyway.
EduHelm is now the first novel treatment to address an underlying cause of Alzheimer’s – the beta-amyloid plaques that accumulate in the brain.
drug endorsed patient and industry group (also the FDA famous “The need for treatment is urgent”) in a statement explaining the agency’s choice. Yet many doctors have expressed concern. A member of the expert committee voted not to recommend approval for Eduhelm in November. resigned since the announcement.
However, the inconsistency of the science and the excessive public debate surrounding the approval of EduHelm has not deterred enthusiasm within the pharmaceutical industry. Instead, it could prompt a new wave of additional treatments in the next few years, which will backfire from approval of EduHelm (though it is controversial whether approval may take place).
“This is great news for investors and pharmaceutical companies working towards new drugs,” says Alison WardA research scientist at the USC Schaefer Center for Health Policy and Economics.
Historically, there have been factors that have made drug development an uphill battle for Alzheimer’s.
First, there is a 17-year history of failure to bring the drug through clinical trials. Even Biogen’s clinical trials for EduHelm were halted in 2019 because it was unclear where they would reach their clinical endpoints (effectively, the trial’s target outcome). Will reach In fact, Aduhlem was approved on the basis of a “surrogate endpoint,” the decline of beta-amyloid, the primary endpoint, not cognitive function.
Tests for Alzheimer’s drugs have also historically been expensive. 2018 paper Alzheimer’s and dementia: translational research and clinical intervention (a magazine run by the Alzheimer’s Association) Estimated cost to develop Alzheimer’s drug was approx. $5.6 billion. By comparison, the average investment required to bring a new drug to market is around $1.3 billion According to an analysis of SEC filings for companies that applied for FDA approval between 2009 and 2018 (though the average cost was about $985 million). Old estimates overestimate the cost of bringing the drug to market $2.8 billion.
For Alzheimer’s specifically, Phase 3 trials are still largely industry-sponsored, but in the past five years, entirely industry-sponsored trials have increased. decreased. Funding through government grants and public-private partnerships has created an i. has made itgrowing part of available funds.
Martin Toler, Founding CEO & CEO alzenAnother company making oral treatments for Alzheimer’s (currently in phase III clinical trials) says attracting other forms of funding was a challenge.
“It was impossible to finance anything,” he says. “It was impossible to be interested in Wall Street because everything was failing one after the other.”
He hopes the recent approval of Eduhelm will significantly change that approach. Already, we’re seeing some increased interest in companies already in phase 3 clinical trials: following the FDA’s announcement, Eli Lilly shares, is also running a phase 3 clinical trial, 10 percent increase.
“I’ve had hundreds of discussions with bankers, investors, associates, pharma, you name it,” Toler says. “Alzheimer’s is Open for Business.”
With renewed interest and what seems like a path to approval at the FDA, the environment for the next generation of Alzheimer’s drugs is ripening. Now, there are almost 130 Phase 3 clinical trials on Alzheimer’s drugs that have either been completed, are active or are recruited.
Toler sees the FDA’s decision based on incomplete data to approve new treatments with impending arrival as “a sign of urgency.”
As pointed out by Ward in a white paper On in-class drug innovation, “follow on” drugs become industry leaders, especially if they demonstrate better safety or efficacy than a drug that first hit the market. The paper argues, however, that drug approval could “pave the way” for more effective drugs in the future.
In the case of Alzheimer’s, it may not be the one drug that dominates, even when more accepted, she notes. Instead, a cadre of new, approved drugs may compliment one another.
“The Way the Medical Community Is Thinking About AD” [Alzheimer’s Disease] Now that it’s probably going to be a combination of drugs or a cocktail of drugs that come together to have true success at delaying progress,” she says.
“If we want to treat AD with a cocktail of drugs, history shows that it is the individually approved drugs that come together to form a cocktail of those drugs.”
There are still some potential pitfalls to consider for future drugs. there is an argument That by having an approved drug available, it could be more difficult to enroll participants in clinical trials, which could slow the pace of drug discovery. In that regard, Ward argues that it will eventually be dwarfed by patients who will consider a possible diagnosis of Alzheimer’s now that there is something to treat it.
There is also a fact that the cost of Eduhelm is high (approx. .) $56,000 For one year’s supply, which will bear the brunt covered by Medicare), and the data remains ambiguous. Those factors may push patients to other drugs, even if they are in clinical trials.
Additionally, there is the question of how well EduHelm actually performs during critical follow-up studies mandated by the FDA as a condition of the drug’s approval. Whether EduHelm can actually slow cognitive decline, as well as help address levels of beta-amyloid from the brain, remains questionable based on current data.
Still, Toller doesn’t see the results of that study as particularly relevant as the industry would have moved on. Biogen CEO Michelle Vounatos has said that he cannot share the results of this test with as many people as possible. nine yearsHowever, he noted that the company will try to provide the data soon.
“By then there will be better drugs,” Toler predicts.
Toler’s Phase 3 Clinical Trial Just Started Dosing This Week, And It’s Scheduled end 2024.
Biogen and Esai will likely Too By then there is another drug ready for evaluation, as two phase 3 clinical trials are scheduled for completion for another beta-amyloid antibody treatment called lecanumab. by 2024 and 2027.
The signal sent by Monday’s approval could be a path, rather than an end, for drugs of the future. The data is incomplete, the cost is high, and the controversy is considerable, but the band-aid has been torn.