Aduhelm, a new, completely revolutionary treatment for the dreaded disease of Alzheimer’s was approved on Wednesday by the United States’ drug regulatory authority the Food and Drug Administration (FDA).
Michel Vounatsos is the CEO of Biogen, Inc. and its Chairman is Stelios Papadopoulos, who created the latest promising drug that may actually end up curing the disease.
In a statement issued by the company, its CEO was effusive. “Today, incredibly inspirational for all of us at Biogen, marks the beginning of a new era for patients, caregivers, and the scientific community engaged in Alzheimer’s research,” Vounatsos declared.
“At Biogen, we are committed more than ever to neuroscience and brain health, to pioneering science and working fearlessly to change patients’ lives. Our focus remains, as always, on their unmet need.”
In an interview with Greek state broadcaster ERT, Papadopoulos stated that the drug’s development “is a huge, but first step in the fight against Alzheimer’s in history.
Aduhelm “first drug to hit biological mechanism” of Alzheimer’s
“A difficult disease and it has presented us with many obstacles to bring medicines to treat it,” he noted, before adding: “The last time a drug was approved was in 2003.”
The Biogen chairman further explained that “This drug is the first to hit the biological mechanism of Alzheimer’s — and not just its symptoms.”
Aricept is another drug on the market that treats the symptoms of the disease, but does not cure it.
“The idea (is) that a serious part of the disease, which in this particular drug can and does help to clear and channel out of the brain,” he explained.
“Our results are exclusively for patients in the early stages of the disease,” Papadopoulos stressed, however.
On Wednesday, the FDA approved Aduhelm for the treatment of Alzheimer’s, which is a debilitating disease affecting 6.2 million Americans. The drug was approved using the “accelerated approval” pathway, which can be implemented in the case of a drug meant to be used in the treatment of a serious or life-threatening illness.
Accelerated approval process only for those that provide “meaningful” advantage over existing drugs
Such a drug must also provide a meaningful therapeutic advantage over any existing treatments already on the market.
According to today’s statement issued by the FDA, accelerated approval can be based on the drug’s effect on an endpoint that is reasonably likely to predict a clinical benefit to patients, with a required post-approval trial to verify that the drug provides the expected clinical benefit.
“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.
“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.”
Alzheimer’s is an irreversible, progressive brain disorder that slowly destroys memory and thinking skills, and eventually, the ability to carry out simple tasks. While the specific causes of Alzheimer’s disease are not fully known, it is characterized by changes in the brain—including amyloid plaques and neurofibrillary, or tau, tangles—that result in loss of neurons and their connections. These changes affect a person’s ability to remember and think.
“First step” in slowing down Alzheimer’s progression
Aduhelm represents a first-of-its-kind treatment approved for Alzheimer’s disease. It is the first new treatment approved for Alzheimer’s since 2003 and is the first therapy that targets the fundamental pathophysiology of the disease.
Researchers evaluated Aduhelm’s efficacy in three separate studies representing a total of 3,482 patients. The studies consisted of double-blind, randomized, placebo-controlled dose-ranging studies in patients with Alzheimer’s disease. Patients receiving the treatment had significant dose-and time-dependent reduction of amyloid beta plaque, while patients in the control arm of the studies had no reduction of amyloid beta plaque.
These results support the accelerated approval of Aduhelm, which is based on the surrogate endpoint of reduction of amyloid beta plaque in the brain—a hallmark of Alzheimer’s disease. Amyloid beta plaque was quantified using positron emission tomography (PET) imaging to estimate the brain levels of amyloid beta plaque in a composite of brain regions expected to be widely affected by Alzheimer’s disease pathology compared to a brain region expected to be spared of such pathology.
The prescribing information for Aduhelm includes a warning for amyloid-related imaging abnormalities (ARIA), which most commonly presents as temporary swelling in areas of the brain that usually resolves over time and does not cause symptoms, though some people may have symptoms such as headache, confusion, dizziness, vision changes, or nausea. Another warning for Aduhelm is for a risk of hypersensitivity reactions, including angioedema and urticaria. The most common side effects of Aduhelm were ARIA, headache, fall, diarrhea, and confusion/delirium/altered mental status/disorientation.
Under the accelerated approval provisions, which provide patients suffering from the disease earlier access to the treatment, the FDA is requiring the company, Biogen, 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.
Aduhelm was granted Fast Track designation, which seeks to expedite the development and review of drugs that are intended to treat serious conditions where initial evidence showed the potential to address an unmet medical need.
Aduhelm is made by the Biogen company, based in Cambridge, Massachusetts.
Vounatsos explained to ERT interviewers today that “This decision was made after two years of analyzing the data we had submitted and which had been collected from many years of studies that began in 2007.”
The chairman further explained that “this drug delays the progression of the disease.
“If patients take the drug in the early stages of the disease, they simply slow down the symptoms. It certainly does not reverse or even stop (it) instantly. It is the first step,” Papadopoulos pointed out.
Asked about the high cost of the new treatment, Papadopoulos stated explained that in the U.S., the annual cost for Aduhelm for each patient will amount to $56,000 dollars.
“Typically, drugs in Europe cost less than in America, and in Greece even less,” he noted, before adding “It will be up to the (Greek) state to decide how to pay for it and the percentage that the patient will participate in.”