For the first time in nearly two decades, the Food and Drug Administration approved a treatment for Alzheimer’s disease.
The FDA gave the go-ahead for aducanumab (marketed as Aduhelm), which is believed to be the first Alzheimer’s drug that targets the cause of the disease rather than the side effects.
The drug, made by Biogen of Cambridge, Massachusetts, was granted “Accelerated approval” Approved by the FDA and shown to clear the amyloid beta plague in the brain – one of the two tell-tale signs of the disease. Aduhelm is the first novel therapy for Alzheimer’s disease to be approved since 2003.
“Alzheimer’s disease is a devastating disease that can have profound effects on the lives of people diagnosed with the disease and their loved ones.” Patrizia Cavazzoni, MD, director of the FDA’s Center for Drug Evaluation and Research, said in a statement Monday. “Currently available therapies only treat symptoms of the disease; This treatment option is the first therapy to target and affect the underlying disease process of Alzheimer’s disease. As we have learned from the fight against cancer, the accelerated approval path can bring therapies to patients faster while promoting more research and innovation. “
Alzheimer’s disease affects 6.2 million Americans, according to the FDA. It is an irreversible, progressive disease of the brain that slowly destroys memory and thinking skills, and then later decreases the ability to do simple tasks.
Michelle Branham, vice president of public policy for the Florida area at Alzheimer’s Association, said the disease is the sixth leading cause of death in the United States and the only cause in the top 10 with no preventive treatment or cure.
Florida is the state with the second highest number of people affected by Alzheimer’s disease, and it is the sixth leading cause of death for its residents.
“(The new drug) treats the disease in ways we’ve never seen before, compared to the currently approved drugs you’re seeing that treat the symptoms of Alzheimer’s and related dementia.” said Branham. “This new type of drug is really a treatment. The therapy not only slows the symptoms but also slows the progression of the disease. This is solemn news for all people with Alzheimer’s and their families. That gives you more quality time, especially if you received an early diagnosis. “
While the specific cause of the disease hasn’t fully been discovered, the FDA said Alzheimer’s is characterized by changes in the brain, including amyloid plaques and neurofibrial (or tau) entanglements that result in the loss of neurons and their connections. Studies show that this new intravenous regimen, which is infused every four weeks, leads to a reduction in amyloid plaques.
“It shows that removing the amyloid from the brain can delay clinical decline in people with Alzheimer’s disease.” said Branham. “It really removes a bit of that sticky amyloid protein that covers the brain and creates brain plaques that are really the hallmark of Alzheimer’s disease.”
Medical professionals, including Branham, hope that in the near future this approval will spark the spark for something bigger when it comes to preventing and stopping dementia-related diseases.
“It’s a new day” Alzheimer’s Association President and CEO Harry Johns said in a statement Monday. “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. “
Maria C. Carillo, Ph.D., Senior Science Officer for the Alzheimer’s Association added: “This FDA drug approval heralds a new era in Alzheimer’s treatment and research. History has shown us that the approval of the first drug in a new category invigorates the field, increases investment in new treatments and stimulates more innovation. We are hopeful and this is the beginning – both for this drug and for better treatments for Alzheimer’s. “
Approval is being reviewed by medical experts and even an FDA advisory committee that rejected the drug after clinical trials and raised concerns about its effectiveness.
“The data contained in the applicant’s application were very complex and left residual uncertainties with regard to the clinical benefit.” Cavazzoni wrote on Monday. “We carefully examined the results of the clinical study, consulted the Advisory Board on Drugs of the Peripheral and Central Nervous System, listened to the perspectives of the patient community and reviewed all relevant data. Ultimately, we opted for the Accelerated Approval Pathway – a pathway designed to give patients with severe illnesses early access to potentially valuable therapies when there is an unmet need and clinical benefit is expected despite some residual uncertainty about that benefit. In determining that the application met the requirements for accelerated approval, the agency concluded that the benefits of Aduhelm for patients with Alzheimer’s disease outweigh the risks of the therapy. “
The drug’s late development program included two Phase 3 clinical trials, one of which showed a reduction in clinical decline, while the second failed to meet the primary endpoint.
“In all studies in which it was evaluated, however, Aduhelm consistently and very convincingly reduced the amount of amyloid plaques in the brain in a dose- and time-dependent manner.” Cavazzoni wrote. “Reducing amyloid plaque is expected to reduce clinical decline.”
She added that removing and reducing amyloid beta plaques in the brain “Almost likely to predict an important benefit for patients.”
Under the expedited approval process, Biogen is required by the FDA to conduct a new randomized controlled clinical trial “to review the clinical benefit of the drug.”
“If the study does not confirm the clinical benefit, the FDA can initiate proceedings to withdraw approval of the drug.” Cavazzoni said.
Side effects of the drug include headache, dizziness, nausea, vomiting, brain swelling, or bleeding. About 40% of the two Phase 3 study participants who received high doses had side effects – most of them were headache, dizziness, or nausea.
“The drug seems to be very well tolerated in clinical studies” said Branham. “Every drug has side effects, we know that. The side effects mentioned – we could argue that in our Alzheimer’s disease world, you’d trade this for more time, more time with your loved one, and that’s quality and more time for memories. I think you need to talk to your doctor about this and make sure that this is a drug that you as a patient can tolerate and know all the side effects that are associated with it and how you personally felt about it.
She argued “If you could have more time with your loved one, more time and quality of life for yourself, wouldn’t you try that? Do not you want that?”
Biogen announced in a press release Monday afternoon that the annual cost of treatment would be $ 56,000 and that the outlay for patients on insurance would vary based on coverage. Treatment also requires additional tests along the way.
“We will do everything in our power to ensure access to the drug and any tests required during the treatment process.” said Branham. “We will do everything we can to remove barriers to entry. This will have the highest priority for us as an association in the future. “
The drug is expected to see high demand in the Sunshine State, which has the fifth oldest median age among the US states.
“Florida really is a ground zero for Alzheimer’s disease” said Branham. “Our leadership has made Alzheimer’s disease a priority since this last legislative term. The governor has put in place a rigorous dementia action plan for the state and a state health improvement plan for Alzheimer’s – the only state with this priority.
“There’s great research going on here in Florida, and it’s exciting. We have to be the gold standard and be at the forefront. “
–Connect with this reporter on Twitter: @haddad_cj
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