"U.S. health regulators approved the first new Alzheimer's drug in nearly two decades, casting aside doubts about the therapy's effectiveness.
The approval Monday of the therapy, which has the molecular name aducanumab and will be sold as Aduhelm, marked a watershed in Alzheimer's drug research after billions of dollars in investment. Maker Biogen Inc. developed the therapy to do what previously approved Alzheimer's medicines can't: slow the memory-robbing march of the disease.
The U.S. Food and Drug Administration, explaining its decision, said scientific evidence indicated Aduhelm reduces a sticky substance in the brain called amyloid -- which is associated with Alzheimer's -- and is likely to produce a benefit to patients.
The drug's sale offers hope to millions of people dealing with Alzheimer's and their caregivers, given the lack of good options for treatment. Yet Aduhelm's impact might be limited. Doctors who said they will prescribe the drug caution it won't help all patients, particularly those with more advanced disease. Some patients eligible for treatment might face $10,000 or more in annual out-of-pocket costs, health insurer Cigna Corp. estimated.
In addition, some doctors said the evidence supporting the drug's efficacy is limited and recommended against its approval.
An outside panel of independent experts convened by the agency rejected supporting the drug, saying Biogen should conduct another large study to prove the drug's benefit before it was approved.
"There is no persuasive evidence to support approval of aducanumab at this time," wrote three of the FDA's independent experts in the Journal of the American Medical Association in March.
Still, the drug is a potential blockbuster for Biogen. Its approval comes at a critical time for the biotech company, which is coping with declining sales and the loss of patent protection for its biggest-selling drug, Tecfidera, a pill for multiple sclerosis.
Biogen has said it expects aducanumab sales to be modest this year and to grow thereafter. Analysts polled by FactSet project sales of $62.7 million in 2021, $603.2 million in 2022 and $1.6 billion in 2023.
Biogen shares soared 38% Monday, adding $16.5 billion in market value. The Nasdaq Biotechnology Index rose 3.6%, its best day since Nov. 4, 2020.
"This historic moment is the culmination of more than a decade of groundbreaking research in the complex field of Alzheimer's disease," said Biogen Chief Executive Michel Vounatsos. "Together with the healthcare community, we are ready to bring this new medicine to patients and begin to address this growing global health crisis."
Biogen priced the newly approved drug higher than analysts expected. The company said it would charge about $56,000 a year per patient.
A preliminary analysis by the Institute for Clinical and Economic Review, a nonprofit research and advisory group, said the drug could be cost-effective at a per patient price of $2,500 to $8,300 a year.
Alzheimer's is a progressive degenerative disease that slowly robs people of their memory and the ability to care for themselves.
About six million people suffer from Alzheimer's in the U.S. Of those, as many as 1.4 million could be eligible to take Aduhelm, according to estimates by Cigna.
There were 121,499 deaths from Alzheimer's in the U.S. in 2019, up 54% from a decade earlier, according to the federal Centers for Disease Control and Prevention. After accounting for age and population growth, per capita deaths grew 23% over the period.
Despite considerable effort and investment, researchers have struggled to find drugs that prove to treat Alzheimer's. One drug after another aiming to slow the progression of the disease has failed in testing.
The FDA last approved a new Alzheimer's drug in 2003. Approved treatments, including the drugs Namenda and Aricept, can help reduce symptoms temporarily but don't change the underlying course of disease, according to regulators.
The FDA granted Aduhelm an accelerated approval, a mechanism for getting drugs to patients faster than the agency otherwise would. The FDA said Biogen would have to conduct a post-approval study to confirm the drug's benefit, and the agency said it could take steps to remove it from the market if the drug doesn't work as intended.
Biogen developed Aduhelm to treat people with mild cases of cognitive impairment or dementia whose brains have accumulated beta amyloid, which many researchers believe plays a significant role in Alzheimer's for many patients.
"The clinical trials for Aduhelm were the first to show that a reduction in these plaques -- a hallmark finding in the brain of patients with Alzheimer's -- is expected to lead to a reduction in the clinical decline of this devastating form of dementia," said Patrizia Cavazzoni, who heads the FDA division that regulates drugs.
Biogen faces hurdles getting the drug to all the patients interested in it, said Richard Isaacson, director of the Alzheimer's Prevention Clinic at Weill Cornell Medicine and New York-Presbyterian hospital, who is a paid consultant to the company.
Unlike other Alzheimer's drugs delivered in pills, Aduhelm requires monthly infusions at a clinic. Patients will require monitoring with an MRI to guard against small brain bleeds, a potential side effect, Dr. Isaacson said.
In clinical trials, 41% of Aduhelm-treated patients had side effects detected by MRIs in which their brains had hemorrhages or an accumulation of fluid, according to the FDA. Most patients didn't suffer any symptoms from the side effects.
The drug also faces skepticism from some doctors over its path to approval.
In 2019, Biogen halted two studies of the drug after determining that they were unlikely to be successful, only to reverse course after reviewing additional data. The company said it would seek approval based on the discontinued studies.
In one of the two studies, patients taking Aduhelm had a 22% reduction in cognitive decline compared with patients taking placebos. The drug failed to show a benefit in the second study, but a detailed breakdown of the results indicated the drug was effective when given at the highest dose for extended periods, Biogen said.
Some FDA officials appeared to agree with Biogen's analysis, giving a positive medical review of the drug during the outside panel's meeting. The agency's statistical expert gave a more downbeat review, citing conflicting trial data.
Dr. Cavazzoni said the FDA decided to approve the drug due to the strong need for Alzheimer's treatment and an expectation that the drug's ability to reduce amyloid will lessen cognitive decline." [1]
In Lithuania where the main assets are stolen by Landsbergiai and their minions, we did not create an economy able to buy such expensive medicines. What could be done in Lithuania today?
"WSJ: What are some preventive measures people can take?
A: Diet. People on vegetarian or Mediterranean diets have cleaner brains in regards to Alzheimer's pathology than those who eat a diet rich in saturated fat. Reducing vascular risks, controlling blood pressure, cholesterol, diabetes, weight -- all of those have been shown to reduce your risk for developing dementia. Be mentally and socially active.
Hearing loss may be one of the biggest potentially reversible factors for Alzheimer's risk. Studies have shown that if you have hearing loss for a long period, it causes shrinkage of brain regions that are very close to the memory centers. So hearing aids and regular hearing tests are very important.
WSJ: What about sleep?
A: We know that sleep is when the brain boosts our immunity and archives our memories. We know that sleep is important for clearing toxic proteins from the brain. All those are crucial. So yes, it is important to get a good night's rest for a healthy memory." [2]
1. FDA Backs Alzheimer's Drug Over Advisory Panel's Doubts
Walker, Joseph. Wall Street Journal, Eastern edition; New York, N.Y. [New York, N.Y]08 June 2021: A.1.
2.
Health & Wellness: How to Lower Alzheimer's Risk --- A top
researcher discusses steps you can take and the advances in diagnosis
and treatment
Ansberry, Clare. Wall Street Journal, Eastern edition; New York, N.Y. [New York, N.Y]08 June 2021: A.11.
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