FDA rejects Cytokinetics heart failure drug

March 3, 2023

The U.S. FDA declined to approve Cytokinetics' oral drug for a type of heart failure, citing lack of sufficient efficacy data.

Earlier this week, the agency issued a Complete Response Letter regarding Cytokinetics' New Drug Application for omecamtiv mecarbil, an investigational, selective, small molecule cardiac myosin activator, for the treatment of heart failure with reduced ejection fraction (HFrEF).

The California-based company had hoped it had the first therapy indicated for HFrEF that directly targets the mechanisms of the heart responsible for contraction, but the FDA thought otherwise. According to Cytokinetics, the FDA communicated that the phase 3 GALACTIC-HF was "not sufficiently persuasive to establish substantial evidence of effectiveness for reducing the risk of heart failure events and cardiovascular death in adults with chronic heart failure with reduced ejection fraction."

The FDA's decision does not come as a surprise. Back in Dec., the agency's Cardiovascular and Renal Drugs Advisory Committee (CRDAC) voted 8 to 3 that the benefits of omecamtiv mecarbil do not outweigh its risks for the treatment of HFrEF.

In the CRL, the agency asked the company to provide results from an additional clinical trial of the drug in order to establish substantial evidence of effectiveness for the treatment of HFrEF. While Cytokinetics says it will request a meeting with FDA in order to understand FDA’s views regarding the CRL and what may be required to support potential approval of omecamtiv mecarbil, the company says it has no plans to conduct an additional clinical trial.

Instead, the company will focus on the development program for aficamten, a next-in-class cardiac myosin inhibitor, currently in phase 3 trials for obstructive hypertrophic cardiomyopathy.