An expert committee that advises the Food and Drug Administration on cardiovascular medicines on Wednesday voted against an experimental and closely watched treatment for heart failure, dealing a blow to its developer, Cytokinetics.
The drug, called omecamtiv mecarbil, is meant to treat a certain kind of heart failure that involves a lessening of the blood pumped through the left ventricle — commonly referred to as reduced ejection fraction. The FDA accepted Cytokinetics’ application in February this year based on results from late-stage clinical testing of the drug.
The advisory committee voted 8 to 3 that the benefits of the drug did not outweigh its risks. While the FDA is not required to follow recommendations from advisors, it often does.
The research and development journey for omecamtiv mecarbil has taken over a decade. Originally, both Cytokinetics and Amgen were working on its development. By 2020, they were ready to submit an application for approval to the FDA following results from a Phase 3 trial that were technically positive.
Yet, while the study, dubbed GALACTIC-HF, met its main goal, the drug’s benefit fell short of expectations. Treatment lowered the risk of cardiovascular death or other heart events by only 8%, below Amgen’s target of 15%. And the risk of cardiovascular death alone among patients who took the drug was not lower than those given placebo.
Soon after, Amgen decided to hand back the rights to its partner while Cytokinetics continued on with the drug’s development.
Lingering concerns from the GALACTIC-HF study seem to remain. The committee’s vote against the drug was based on their view of its relatively small treatment effect, the lack of a mortality benefit and other questions around safety, according to a research note from Salim Syed, an analyst at the investment firm Mizuho Securities. Syed also wrote that an additional study would likely be needed to prove its benefit.
Following the negative committee vote, some analysts expect investors will turn their attention toward the company’s other heart disease drug, called aficamten, which is currently in a Phase 3 trial.
“We plan to engage constructively with FDA as it completes its review of the application for omecamtiv mecarbil,” said Cytokinetics CEO Robert Blum, in a statement.
The FDA is set to make a decision on the drug by February 28 next year.