Alzheimer’s Drug Poses a Dilemma For The F.D.A.

Current U.S. The Alzheimer’s disease therapy Aduhelm (aducanumab) was recently approved by the Food and Drug Administration (FDA). This illness affects 6.2 million Americans. The FDA chose to fast-track Aduhelm’s approval because the medicine represents a significant improvement in patient outcomes above standard care for patients with serious or life-threatening illnesses.

Alzheimer’s Drug Poses a Dilemma For The F.D.A.

With a needed post-approval trial to confirm that the medicine provides the projected clinical benefit to patients, accelerated approval can be based on the drug’s effect on a surrogate endpoint that is reasonably likely to predict a therapeutic benefit to patients.

Alzheimer’s Disease is a Severe Condition

Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research, said, “Alzheimer’s disease is a severe condition that can have a tremendous impact on the lives of persons diagnosed with the disease and their loved ones.” This is the first medicine to target and alter the underlying disease process of Alzheimer’s, whereas current treatments just address the symptoms of the disease. The experience gained in the war against cancer has taught us that the accelerated approval process can hasten the delivery of medicines to patients and encourage further exploration and development of new approaches.

Alzheimer’s is a degenerative brain disease that robs sufferers of their capacity to remember things, think clearly, and do even the most menial of tasks over time. Amyloid plaques and neurofibrillary, or tau, tangles are two hallmarks of Alzheimer’s disease, which is also defined by the death of neurons and the disruption of their connections. Memory and cognition are impacted by these shifts.

The approval of Aduhelm is a historic first in the treatment of Alzheimer’s disease. It is the first medicine to tackle the fundamental biology of Alzheimer’s disease, and the first novel treatment licenced for the disease since 2003.

In three studies including 3,482 patients, researchers analysed the effectiveness of Aduhelm. Patients with Alzheimer’s disease participated in placebo-controlled, randomised, dose-ranging experiments. In contrast to the control group, patients who received the therapy saw a significant dose- and time-dependent reduction in amyloid beta plaque.

Hhallmark of Alzheimer’s Disease

The reduction of amyloid beta plaque in the brain is a hallmark of Alzheimer’s disease, and these findings lend support to the speedy approval of Aduhelm based on this surrogate objective. To quantify the brain levels of amyloid beta plaque in a composite of brain regions believed to be widely affected by Alzheimer’s disease pathology and a brain region expected to be spared from such pathology, positron emission tomography (PET) imaging was used.

Amyloid-related imaging abnormalities (ARIA) are cautioned against in the Aduhelm prescribing information. ARIA most often manifest as temporary swelling in areas of the brain, which usually resolves over time and does not cause symptoms, though some people may experience symptoms like headache, confusion, dizziness, vision changes, or nausea. In addition, Aduhelm comes with a warning that it may cause hypersensitivity reactions such angioedema and urticaria. Aduhelm’s most common adverse reactions included ARIA, headache, falling, diarrhoea, and altered mental status (such as delirium or confusion).

Last Words

The FDA is requiring Biogen to conduct a new randomised, controlled clinical trial to prove the drug’s clinical benefit in order to receive accelerated approval, which would allow patients suffering from the condition to have earlier access to the medication. The FDA may begin actions to revoke the drug’s approval if the trial fails to confirm clinical benefit.

Fast Track designation was given to Aduhelm in an effort to speed up the development and review of the drug for the treatment of serious illnesses where preliminary evidence suggested the therapy could meet an unmet medical need.

Biogen, located in Cambridge, MA, is the company responsible for producing Aduhelm.