FDA Grants Gantenerumab a Breakthrough Therapy Designation for Potential Treatment of Alzheimer Disease

The Food and Drug Administration (FDA) granted a breakthrough therapy designation for gantenerumab (Genentech, San Francisco, CA) for potential treatment of Alzheimer disease (AD). The designation is based on data showing gantenerumab significantly reduced brain amyloid plaque, a pathologic hallmark of AD, in ongoing open-label extension trials. Gantenerumab is also being studied in 2 ongoing parallel global placebo-controlled and randomized phase 3 trials, GRADUATE 1 and 2 (NCT03444870 and NCT03443973). 

“For more than a decade, we’ve been committed to advancing the science of Alzheimer as well as our investigational medicine gantenerumab, and we look forward to delivering a comprehensive and robust data set that furthers our collective understanding of this devastating disease,” said Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development. “This breakthrough therapy designation reinforces our confidence in gantenerumab, which would be the first subcutaneous medicine for the treatment of Alzheimer disease with the potential for at-home administration.”

The pivotal GRADUATE trials are evaluating the effect of gantenerumab on amyloid load and downstream biomarkers of disease progression in 2,000 individuals with early (prodromal-to-mild) AD. The studies assess monthly target dose of 1,020 mg with an optimized titration for more than 2 years with an expected completion in the second half of 2022. The titration aims to maximize exposure and minimize dose interruption for better detection of clinical benefit. 

Gantenerumab is an investigational IgG1 antibody designed to bind to aggregated forms of amyloid β and remove brain amyloid plaques. Gantenerumab significantly lowered brain amyloid plaques in patients with sporadic AD in the SCarlet RoAD and Marguerite RoAD open-label extension studies (OLEs) and in participants in the DIAN-TU-001 study (NCT01760005).