Aficamten hits both endpoints in nHCM trial

On Friday, Cytokinetics put the full pivotal-study results for aficamten on the table. In patients with non-obstructive hypertrophic cardiomyopathy, or nHCM—an inherited heart condition—the drug met both endpoints, improving how patients felt and their exercise capacity.

That result moves aficamten toward a regulatory test, not across the finish line. Cytokinetics plans to submit the drug to the Food and Drug Administration (FDA) by the end of the year. The agency has not yet decided whether to approve it.

The company’s timeline would mark a sharp contrast with its history. Cytokinetics took 27 years to win its first regulatory approval; the second could come much faster if aficamten clears the FDA review. If approved, the drug would be the first treatment specifically cleared for nHCM.

For patients, that is the concrete possibility: a treatment specifically cleared for this particular inherited heart condition. The result is still limited to the pivotal study, and STAT reported that some experts have described the benefits as limited.

The next step is therefore defined but uncertain: Cytokinetics has a planned filing and positive trial endpoints, but it does not yet have an FDA-cleared treatment.