For the first time since October of 2003, the U.S. Food and Drug Administration has Approved a New Treatment for Alzheimer's Disease.

For the first time since October of 2003, the U.S. Food and Drug Administration has approved a new treatment for Alzheimer's disease. It is a drug called Aduhelm. The drug is credited with slowing the progression of Alzheimer's disease itself, rather than simply decreasing the intensity of the symptoms.

After an initial titration period, the drug is given as an intravenous infusion about every four weeks. If the patient is able to overcome some possible allergic reactions, they must bear the list price of about $56,000. a year for the drug.

The manufacturer of the drug, Biogen is making available support service coordinators to assist patients and their caregivers or family members with the treatment and its cost. These coordinators can be reached at 1-833-425-9360.

Biogen is also working with CVS Health’s Project Health, to aid uninsured and underinsurance Americans with cognitive screenings that could help in diagnosing Alzheimer’s. It is also working with the Veterans Health Administration to set up a program supporting access there.

Some uncertainty remains, however. The FDA approved the drug under what is called the "accelerated approval pathway." This is intended to provide earlier access to therapies that are expected to benefit patients with serious disease, even if there remains some uncertainty regarding that benefit.

The drug itself is described as a human-made antibody designed to remove toxic clumps of the protein beta-amyloid, which are thought to cause the death of nerve cells in the brains of patients with Alzheimer's. However, the drug's trials did not go entirely as expected. Phase 1b of the trial suggested that the drug could safely slow cognitive decline and also remove beta-amyloid plaques as intended. But then Phase 3 trials indicated that the drug, given as a monthly intravenous infusion, was unlikely to benefit patients, and the drug trials were discontinued. But then new data became available which seemed to indicate the drug actually did improve cognition and function, including memory, orientation, language and activities of daily living. However, it benefitted those given a high dose and those in the early stages of the disease.

Based on this new and favorable analysis, and in conjunction with the positive results in the earlier trials, the drug manufacturer sought regulatory approval in the U.S. At first, the FDA advisory committee voted that available clinical data did not support Aduhelm as an effective Alzheimer's treatment. Some experts suggested a new Phase 3 trial at the higher doses prior to an approval. Neurologists with the Mayo Clinic and Stanford University did not agree with early approval.

However, the FDA stated: “We are well-aware of the attention surrounding this approval. … With treatment for a serious, life-threatening disease in the balance, it makes sense that so many people were following the outcome of this review,” the FDA also acknowledged that the clinical trial data on the therapy “were highly complex and left residual uncertainties regarding clinical benefit.” However, the FDA said that although the clinical trials were not conclusive on the therapy's effect on cognition and function, they clearly showed that the drug reduced levels of beta-amyloid plaques, and that data helped formed the basis for the FDA to grant accelerated approval of the drug.

“Treatment with Aduhelm was clearly shown in all trials to substantially reduce amyloid-beta plaques. This reduction in plaques is reasonably likely to result in clinical benefit,” the FDA wrote.

As a stipulation of the approval, the medication’s manufacturers will be required to conduct additional clinical testing to verify the anticipated clinical benefit, known as Phase 4 confirmatory trials. If data from these trials do not verify the anticipated benefit, the FDA could remove Aduhelm from the market.

The drug has not yet been approved in the European Union or Japan and those applications are still under review.

Would you try this drug? Why or why not?

tags: medical, new therapies