Novel Medical Therapeutics for Hypertrophic Cardiomyopathy
Drugs used in the treatment of HCM are largely off-label. In addition, their efficacy is variable, with none approaching a high level of consistent efficacy. Accordingly, increasingly symptomatic patients move on to invasive therapies, including septal reduction therapy and heart transplantation. HCM has been given orphan drug discovery status, spurring multiple companies to invest in novel pharmacotherapies in this space. Three drugs have reached development, with two recently halted. The remaining drug, mavacamten (Myokardia, Inc.), is approaching its pivotal trial after encouraging results in pilot trials on patients with obstructive HCM. Further research is needed, but it seems likely that new medications with greater efficacy will be available commercially within the next decade.
KeywordsNovel drugs Orphan drug Mavacamten Perhexiline Eleclazine
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