The FDA grants accelerated approval for Aduhelm (aducanumab), the first-of-its-kind treatment since 2003 for Alzheimer’s disease. The FDA approved the drug for marketing, and requires Biogen to conduct another study to prove that it is effective. The drug will be available for a prescription, while another clinical study will be underway.
According to the Alzheimers’s Association, more than 6 million Americans live with Alzheimer’s. This is expected to rise to nearly 13 million by 2050. Alzheimer’s is a progressive brain disease, and it is the most common cause of dementia’s memory loss that interferes with daily life. Alzheimer’s begins with memory loss (typically affecting the learning area of the brain) and advances to severe symptoms of memory failure, difficulty speaking, swallowing and walking. Alzheimer’s has no cure, but Aduhelm (aducanumab) is approved to be a new treatment to slow clinical decline and improve quality of life.
The FDA approval was based on the clinical study results that aducanumab reduced amyloid beta plaques, a measureable biomarker predicting a reduction in clinical decline. Aducanumab was studied in two Phase 3 clinical trials, EMERGE (Study 1) and ENGAGE (Study 2), in patients with confirmed amyloid pathology in early stages of Alzheimer’s disease. Aducanumab was also evaluated in Phase 1b study, PRIME (Study 3), a double-blind, randomized, placebo-controlled, dose-ranging clinical trial. Across all three studies with over 3,000 patients who received at least one dose, aducanumab consistently showed a lowering of amyloid beta plaques ranging from 59 percent [p<0.0001] in ENGAGE, 71 percent [p<0.0001] in EMERGE, and 61 percent [p<0.0001] in PRIME.
The approval does not come without controversy. In fact three members of a Food and Drug Administration advisory committee have now resigned in protest. Resignations include Harvard Medical School professor Aaron Kesselheim, Mayo Clinic neurologist David Knopmanat, and Washington University in St. Louis neurologist Joel Perlmutter.
There is debate about whether the evidence was sufficient for FDA approval. The most frequently reported adverse reaction was the MRI detection of Amyloid Related Imaging Abnormalities, or “ARIA” in 41 percent of patients compared to 10 percent of patients on placebo. The affect of aducanumab in the clinical trials found that some patients experienced painful brain swelling. The most common symptom in patients with ARIA was headache.
The price tag on Aduhelm (aducanumab) was much higher than expected. Biogen said it is charging $56,000 for an annual course of the new treatment.
There are other pipeline drugs being studied to treat Alzeheimer’s, such as Eli Lilly’s donanemab and and Denmark’s Novo Nordisk’s semaglutide. Perhaps these treatments, along with Aduhelm (aducanumab), will reach the market for this entirely unmet need, and improve the lives of millions of people.