Mavacamten, a novel agent for symptomatic HOCM, is under review by the US Food and Drug Administration, with a decision to
approve the treatment expected in April 2022. Mavacamten reduces adenosine triphosphatase activity in cardiac myosin heavy chain, one of
the proteins in heart muscle cells, and reduces the contraction of the heart that can contribute to obstruction. The Institute for Clinical and Economic Review (ICER) conducted a systematic literature review and cost-effectiveness analysis to evaluate health and economic outcomes of mavacamten in patients with symptomatic HOCM. Evaluations were also conducted of the available evidence on septal reduction procedures and on treatment with disopyramide. Complete details of ICER’s systematic literature search and protocol, as well as the methodology and model structure for the economic evaluation, are available on ICER’s website at https://icer.org/hypertrophic-cardiomyopathy-2021/. Here, we present the summary of our findings and highlights of the policy discussion with key stakeholders held at a public meeting of the California Technology Assessment Forum (CTAF) on October 22, 2021.
