FDA Grants Empagliflozin Breakthrough Therapy Status for Heart Failure With Preserved Ejection Fraction

$FDA Grants Empagliflozin Breakthrough Therapy Status for Heart Failure With Preserved Ejection Fraction. he U.S. Food and Drug Administration (FDA) recently granted the breakthrough therapy designation for empagliflozin (Jardiance) as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF).$

October 7, 2021 – The U.S. Food and Drug Administration (FDA) recently granted the breakthrough therapy designation for empagliflozin (Jardiance) as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF).

The decision is based on results from the landmark EMPEROR-Preserved phase III trial, in which empagliflozin demonstrated a 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction compared with placebo. The benefit was independent of ejection fraction or diabetes status. Results from EMPEROR-Preserved were presented at the 2021 European Society of Cardiology (ESC) Congress 2021 Aug. 27 and published in The New England Journal of Medicine.[1]

HFpEF accounts for approximately half of the more than 6 million heart failure cases in the U.S. No currently approved treatments have been clinically proven to significantly improve outcomes specifically for people with HFpEF.

“This Breakthrough Therapy designation underscores the potential of Jardiance to help fill a critical need for a clinically proven treatment for people with this highly prevalent, difficult-to-treat condition,” said Mohamed Eid, M.D., M.P.H., M.H.A., vice president, clinical development and medical affairs, cardio-metabolism and respiratory medicine, Boehringer Ingelheim Pharmaceuticals. “Following the recent FDA approval of Jardiance for heart failure with reduced ejection fraction, this is another important milestone supporting the potential of Jardiance as the first therapy clinically proven to improve outcomes across the full heart failure spectrum.”

According to the FDA, the breakthrough therapy designation is designed to expedite the development and review of therapies that are intended to treat a serious condition where preliminary clinical evidence indicates potentially substantial improvement over available therapies on a clinically significant endpoint.

“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, M.D., Ph.D., vice president, product development, Lilly. “Together with our Boehringer Ingelheim partners, we look forward to working closely with the FDA through this accelerated process to potentially bring Jardiance to adults with heart failure with preserved ejection fraction as soon as possible.”

The FDA previously granted Fast Track designation for the development of empagliflozin to reduce the risk of cardiovascular death and hospitalization for heart failure. This designation is for the EMPEROR program, which consists of the EMPEROR-Reduced and EMPEROR-Preserved trials. The EMPEROR-Reduced results formed the basis of the recent FDA approval for heart failure with reduced ejection fraction. empagliflozin is not indicated for the treatment of HFpEF.

The drug has been jointly developed by Boehringer Ingelheim and Eli Lilly and Company.

About the EMPEROR-Preserved Trial

EMPEROR-Preserved (NCT03057951) was a phase III international, randomized, double‐blind trial that enrolled 5,988 adults with and without type 2 diabetes. All participants had heart failure (New York Heart Association [NYHA] functional class II, III or IV) and preserved ejection fraction (left ventricular ejection fraction > 40%).

Participants were randomized to once-daily empagliflozin 10 mg (n=2997) or placebo (n=2991), on top of treatment with guideline-directed heart failure therapy. Median follow-up time was 26.2 months. The composite primary endpoint was defined as time to first event of cardiovascular death or hospitalization for heart failure.

For more information: www.jardiance.com