The US FDA approved Biogen's Aduhelm (aducanumab) for Alzheimer's disease. It is the first approved product for AD since 2003 and acts by targeting the pathophysiology of the disease.
The approval is based on three double-blind placebo-controlled trials. The drug demonstrated dose-and-time dependent reduction in amyloid-beta plaque. The company has submitted EMERGE (Study 1) and ENGAGE (Study 2) studies; patients with confirmed presence of amyloid pathology in the early stages were included in the trials. Amyloid-beta plaques were reduced by 59% in EMERGE trial and by 71% in ENGAGE trial.
FDA complete approval will be based on the benefit demonstrated in the confirmatory trials.
The drug received the accelerated approval –approval given for serious deliberating diseases and therapies that significantly benefit the existing drugs.
Biogen initiatives to promote the drug
Biogen and Eisai initiated a range of services to promote the drug. The company announced personal Biogen Support Service Coordinators to give one-on-one support – to give information to patients on Alzheimer's disease and Aduhelm treatment. The company also collaborated with Labcorp and Mayo Clinic Laboratories to help to diagnose the condition. In addition, the company is making deals with the Veterans Health Administration to provide access to veterans.
ICER criticized the efficacy and safety of Aduhelm
However, ICER has criticized FDA for approving the drug. ICER commented that by approving the drug with unproven efficacy with known harms, the FDA failed to safeguard patients' safety. The organization stated that the current evidence does not justify benefits. Further, ICER cited that the drug has severe side effects. Instead of giving preference to patient-centered outcomes of efficacy, ICER slammed FDA for considering surrogate endpoints. It emphasized that other medications have successfully removed amyloid from the brain yet were not successful in helping the patients. It highlighted that the FDA approved for all patients while the studies included only patients with mild cognitive impairment and mild dementia.
Is Aduhelm priced too high?
The annual cost of treatment with Aduhelm is $56,000. The CEO of Biogen, Michel Vounatsos, justified the high price. However, ICER commented that the drug should be ideally priced between $2,500-$8,300. Even in the best-case scenario, ICER commented that the drug price should be between $11,100-$23,100. A drug that halts dementia only justifies the price of $56,000, and ICER commented that Aduhelm was priced far higher than the favorable price.