Investigational Medication

Overview: There are ongoing studies in drugs for treating HCM or its symptoms. These are called investigational or experimental drugs. 

In this section we are going to talk about what Investigational medications are and their pathway to consumers.  


Investigational drugs under clinical investigation.

As of Summer 2021, there are several new drugs under investigation for use in HCM. Below is a list of several medications at different stages of discovery, trial or submission to the FDA (Food and Drug Administration - USA)

Mavacamten: Indications: HOCM: New Drug Application (NDA) was submitted in 2021 with a Prescription Drug User Fee Act (PDUFA) goal date of January 28, 2022.  This means the data from trials in those with HOCM results were submitted to the FDA for review.    The explanation of the mechanism and use recommendations will be addressed on a page soon to be added to the site. Sponsor: MyoKardia/Bristol Myers Squibb

CK-274: Indication: HOCM:  Phase 2 clinical trial.  This agent is in the same class as Mavacamten with slight differences in the action.   Clinical trials are ongoing. Sponsor: Cytokinetics

IMB-101 Indication: HCM: Phase 2 study in non-obstructive HCM.  For non-obstructive HCM patients with preserved systolic function (ejection fraction (EF) ≥50%) who develop symptoms refractory to drug therapy, heart transplant remains the only definitive long-term management option. Sponsor: Imbria Pharmaceuticals

Additional content on these trials and other investigational innovations will be added in the next few weeks. 


HCMA 6/2021