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Alzheimer’s drug gets approval

By CJ HADDAD - | Jun 10, 2021

For the first time in nearly two decades, the Food and Drug Administration has approved a treatment for Alzheimer’s disease.

On Monday, the FDA gave the green light to aducanumab (marketed as Aduhelm), considered the first Alzheimer’s drug that targets the cause of the disease, not the side effects.

Created by Biogen out of Cambridge, Massachusetts, the drug was granted “accelerated approval” designation by the FDA and has shown to eliminate amyloid beta plague in the brain — one of the two telltale signs of the disease. Aduhelm is the first novel therapy approved for Alzheimer’s disease since 2003.

“Alzheimer’s disease is a devastating illness that can have a profound impact on the lives of people diagnosed with the disease as well as their loved ones,” said Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research 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. As we have learned from the fight against cancer, the accelerated approval pathway can bring therapies to patients faster while spurring more research and innovation.”

According to the FDA, Alzheimer’s disease affects 6.2 million Americans. It is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills and down the road, diminishes the ability to complete simple tasks.

Michelle Branham, Florida Region vice president of Public Policy for the Alzheimer’s Association, said the disease is the sixth leading cause of death in the U.S., and the only cause in the top 10 without a prevention treatment or cure.

Florida ranks as the state with the second-highest number of individuals impacted by Alzheimer’s disease and it is the sixth leading cause of death for residents.

“(The new drug) addresses the disease in a way we’ve never seen it addressed before, compared to the currently-approved drugs you see that are managing symptoms of Alzheimer’s and related dementia,” Branham said. “This new type of drug is really a treatment. The therapy slows the progression of the disease rather than only addressing the symptoms. That is celebratory news for all people living with Alzheimer’s and their loved ones. It gives more quality time, especially if you’ve received an early diagnosis.”

While the specific cause for the disease has not been fully discovered, the FDA said Alzheimer’s is characterized by changes in the brain, including amyloid plaques and neurofibrially (or tau), tangles, that result in the loss of neurons and their connections. Trials show this new intravenous method of treatment, which would be infused once every four weeks, results in the reduction of amyloid plaques.

“It demonstrates that removing the amyloid from the brain may delay the clinical decline in people with Alzheimer’s disease,” Branham said. “It’s really clearing a bit of that sticky amyloid protein that covers the brain and creates brain plaques that are really the hallmark of Alzheimer’s disease.”

Those in the medical field, including Branham, hope this approval becomes the spark for something bigger in the near future when it comes to preventing and stalling dementia-related disease.

“It is a new day,” said Alzheimer’s Association President and CEO Harry Johns in a statement Monday. “This approval allows people living with Alzheimer’s more time to live better. For families it means being able to hold on to their loved ones longer. It is about reinvigorating scientists and companies in the fight against this scourge of a disease. It is about hope.”

Maria C. Carillo, Ph.D, Alzheimer’s Association chief science added, “This FDA drug approval ushers in a new era in Alzheimer’s treatment and research. History has shown us that approvals of the first drug in a new category invigorates the field, increases investments in new treatments and encourages greater innovation. We are hopeful and this is the beginning — both for this drug and for better treatments for Alzheimer’s.”

The approval does come with some scrutiny from medical experts and even an FDA advisory committee, who declined to endorse the drug after clinical trials, raising concerns about effectiveness.

“The data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit,” Cavazzoni wrote Monday. “We examined the clinical trial findings with a fine-tooth comb, we solicited input from the Peripheral and Central Nervous System Drugs Advisory Committee, we listened to the perspectives of the patient community, and we reviewed all relevant data. We ultimately decided to use the Accelerated Approval pathway–a pathway intended to provide earlier access to potentially valuable therapies for patients with serious diseases where there is an unmet need, and where there is an expectation of clinical benefit despite some residual uncertainty regarding 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 outweighed the risks of the therapy.”

The late-stage development program from the drug involved a pair of phase 3 clinical trials, with one showing a reduction in clinical decline while the second did not meet the primary endpoint.

“In all studies in which it was evaluated, however, Aduhelm consistently and very convincingly reduced the level of amyloid plaques in the brain in a dose- and time-dependent fashion,” Cavazzoni wrote. “It is expected that the reduction in amyloid plaque will result in a reduction in clinical decline.”

She added that the removal and reduction of amyloid beta plaques in the brain is “reasonably likely to predict important benefits to patients.”

Under the accelerated approval provision, Biogen is required by the FDA to conduct a new, randomized controlled clinical trial “to verify the drug’s clinical benefit.”

“If the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug,” Cavazzoni stated.

Side effects of the drug include headaches, dizziness, nausea, vomiting, brain swelling or bleeding. Roughly 40% of the pair of phase 3 trial participants receiving high doses experienced some side effect — the majority experiencing a headache, dizziness or nausea.

“The drug seems to be very well tolerated in clinical trials,” Branham said. “Every drug has side-effects, we know that. The side effects mentioned — we could argue in our world of Alzheimer’s you would trade those for more time, more time with your loved one, and that’s quality, and more time for memories. I think it’s something that you do have to talk with your clinician about and make sure this would be a drug you could tolerate as a patient and knew all of the side-effects going into it and how you felt about it personally.”

She argued, “If you could get more time with your loved one, more time and quality of life for yourself, wouldn’t you try that? Wouldn’t you want that?”

Biogen, in a release Monday afternoon, stated the yearly cost of the treatment would be $56,000 and that the out-of-pocket cost for patients with insurance will vary depending on coverage. The treatment will also require additional testing along the way.

“We’re going to do everything in our power to ensure access to the drug and any tests needed during the treatment process,” Branham said. “We will put all of our efforts an advocacy into eliminating barriers to access. That will be our highest priority as an association going forward.”

The drug is expected to be in great demand in the Sunshine State, which has the fifth-oldest average age among the U.S. states.

“Florida really is a ground zero for Alzheimer’s disease,” Branham said. “Our leadership has prioritized Alzheimer’s from this past legislative session. The governor put together a rigorous dementia action plan for the state and a state health improvement plan priority for Alzheimer’s — the only state with that priority.

“There’s some great research being done here in Florida and that’s exciting. We need to be the gold standard and at the front of the pack.”

–Connect with this reporter on Twitter: @haddad_cj