Cytokinetics bags FDA breakthrough therapy status for aficamten in oHCM


Cytokinetics has secured breakthrough therapy designation from the Food and Drug Administration (FDA) for aficamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

According to the -based biopharma company, Aficamten is a cardiac myosin inhibitor, which is being developed for the potential treatment of hypertrophic cardiomyopathy.

In patients with hypertrophic cardiomyopathy, the heart muscle becomes abnormally thick.

The breakthrough therapy designation for aficamten has been driven by the findings from cohorts 1 and 2 of the phase 2 REDWOOD-HCM clinical trial in patients having symptomatic oHCM. As per the results, treatment with aficamten for a period of 10 weeks delivered statistically significant reductions from baseline in comparison to placebo in the average resting left ventricular outflow tract pressure gradient as well as the average post-Valsalva LVOT-G.

With the breakthrough therapy designation, aficamten qualifies for all the features of fast track designation, thorough guidance on implementing an efficient drug development program, and organizational commitment with the involvement of senior managers.

Fady I. Malik — Cytokinetics Executive Vice President of Research and Development said: “We are pleased that the FDA has granted Breakthrough Therapy Designation for aficamten for the treatment of symptomatic oHCM, a disease associated with myriad symptoms and functional limitations.

“The results of REDWOOD-HCM met our high expectations supporting the potential role of aficamten as an innovative therapy to address the hypercontractility that underlies oHCM.

“With start-up activities underway in SEQUOIA-HCM, our Phase 3 clinical trial of aficamten, we look forward to its further development and engaging with FDA as may hopefully benefit patients whose lives are severely impacted by this disease.”

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