Today the U.S. Food and Drug Administration approved Aduhelm (aducanumab) for the treatment of Alzheimer’s, a debilitating disease that affects 6.2 million Americans. Aduhelm was approved on the accelerated path that can be used for a drug for a serious or life-threatening illness that offers a significant therapeutic advantage over existing treatments. Expedited approval may be based on the drug’s action on a surrogate endpoint that is reasonably likely to predict clinical benefit for patients, requiring a post-approval study to confirm that the drug provides the expected clinical benefit .
“Alzheimer’s disease is a devastating disease that can have a profound impact on the lives of people diagnosed with the disease and their loved ones,” said Patrizia Cavazzoni, MD, 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 influence 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 is an irreversible, progressive disease of the brain that slowly destroys memory and the ability to think, and ultimately the ability to perform simple tasks. Although the specific causes of Alzheimer’s disease are not fully understood, it is characterized by changes in the brain – including amyloid plaques and neurofibrillary tangles or tau – that result in the loss of neurons and their connections. These changes affect a person’s ability to remember and think.
Aduhelm is the first drug of its kind to be approved for Alzheimer’s disease. It is the first new treatment approved for Alzheimer’s since 2003 and the first therapy to target the basic pathophysiology of the disease.
The researchers evaluated the effectiveness of Aduhelm in three separate studies involving a total of 3,482 patients. The studies consisted of double-blind, randomized, placebo-controlled dose-range studies in patients with Alzheimer’s disease. Patients who received the treatment showed a significant dose- and time-dependent reduction in amyloid beta plaques, while the patients in the control arm of the studies showed no reduction in amyloid beta plaques.
These results support the accelerated approval of Aduhelm, which is based on the surrogate endpoint of reducing amyloid beta plaque in the brain – a hallmark of Alzheimer’s disease. Amyloid beta plaque was quantified using positron emission tomography (PET) to determine brain levels of amyloid beta plaque in a combination of brain regions expected to be severely affected by Alzheimer’s disease Comparison to a brain region that is expected to be spared from such a pathology, quantified.
The prescribing information for Aduhelm includes a warning for amyloid-related imaging disorder (ARIA), which is most common as a temporary swelling in areas of the brain that usually resolve over time and cause no symptoms, although some people may have symptoms such as headaches , Confusion, dizziness, blurred vision, or nausea. Another warning to Aduhelm is the risk of hypersensitivity reactions, including angioedema and urticaria. The most common side effects with Aduhelm were ARIA, headache, fall, diarrhea and confusion / delirium / altered mental status / disorientation.
As part of the accelerated approval process, which will give patients with the disease earlier access to treatment, the FDA is asking Biogen to conduct a new randomized, controlled clinical trial to evaluate the drug’s clinical utility. If the study does not confirm the clinical benefit, the FDA can initiate a disapproval of the drug.
Aduhelm has been awarded Fast Track status, which aims to expedite the development and review of drugs used to treat serious medical conditions where early evidence showed the potential to meet an unmet medical need.
Aduhelm is manufactured by Biogen of Cambridge, Massachusetts.
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The FDA, a US Department of Health agency, protects public health by ensuring the safety, efficacy, and safety of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency is also responsible for the safety of our country’s food supplies, cosmetics, dietary supplements, products that emit electronic radiation, and the regulation of tobacco products.
