On 24 June, Eli Lilly has announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for donanemab, an antibody that targets a form of modified amyloid protein, for the treatment of Alzheimer's disease (AD).
The Breakthrough Therapy designation aims to expedite the development and review of drugs that are intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over already available therapies that have received full FDA approval.
The FDA agency based this decision on a randomised, placebo-controlled and double-blind Phase II study evaluating the safety and efficacy of donanemab in people with early symptomatic AD. This TRAILBLAZER-ALZ study enrolled 272 participants and showed a significantly slower decline in cognition and daily function compared to placebo. Donanemab met the primary endpoint of change from baseline to 76 weeks in the Integrated AD Rating Scale (iADRS). In addition, the study reported that donanemab showed consistent improvements in secondary endpoints measuring cognition and function as compared to placebo. Donanemab is also being evaluated in an ongoing randomised, placebo-controlled, double-blind and multi-center Phase III study, named TRAILBLAZER-ALZ 2, to evaluate the safety, tolerability and efficacy of donanemab in people with early symptomatic AD.
