The Food and Drug Administration (FDA) granted Breakthrough Therapy designation to empagliflozin for the treatment of adults with heart failure with preserved ejection fraction.
The designation is based on data from the randomized, double-blind, phase 3 EMPEROR-Preserved trial (ClinicalTrials.gov Identifier: NCT03057951), which compared the efficacy and safety of empagliflozin with placebo in adults with heart failure with preserved ejection fraction, with and without diabetes. Patients were randomly assigned 1:1 to receive either empagliflozin 10mg once daily (n=2997) or placebo (n=2991), in addition to standard of care for up to 38 months.
Results from the trial showed that treatment with empagliflozin met the primary composite endpoint reducing the relative risk of cardiovascular death or hospitalization for heart failure by 21%. The reduction was mainly related to a lower risk of hospitalization for heart failure. Treatment with empagliflozin was also associated with a lower risk of first and recurrent hospitalization for heart failure and significantly slowed kidney function decline. The safety profile of empagliflozin was consistent with that observed in previous findings.
“Given the scarcity of treatment options for this debilitating condition, the benefits demonstrated in the EMPEROR-Preserved trial constituted a major clinical breakthrough,” said Jeff Emmick, MD, PhD, vice president, Product Development, Lilly.
Empagliflozin is marketed under the trade name Jardiance®. In August 2021, the FDA approved Jardiance to reduce the risk of cardiovascular death and hospitalization for heart failure and to slow kidney function decline in adults with chronic heart failure with reduced ejection fraction, including those with and without type 2 diabetes.
The approval was based on data from the phase 3 EMPEROR-Reduced trial (ClinicalTrials.gov Identifier: NCT03057977), which demonstrated that treatment with empagliflozin reduced the combined relative risk of cardiovascular death or hospitalization for heart failure by 25% compared with placebo in adults with heart failure with reduced ejection fraction.
Empagliflozin receives Breakthrough Therapy designation in US for heart failure with preserved ejection fraction (HFpEF). News release. Eli Lilly and Company. Accessed September 9, 2021. https://www.prnewswire.com/news-releases/fda-grants-jardiance-breakthrough-therapy-designation-for-heart-failure-with-preserved-ejection-fraction-301372022.html.