FDA approved Mavacamten (Camzyos from Bristol-Myers Squibb) for HOCM for children (May 2022).
Mavacamten is allosteric inhibitor of Myosin
Explorer-HCM trial data showed
(i) improved in peak VO2 and
(ii) stabilization or improvement in NYHA function class
compared to placebo.
Long-term extension study showed that benefits lasted at 1-year follow up. There was improvement in QoL reported by patients.
Valor-HCM - addition of Mavacamten to maximally-tolerated medical management, reduced the need for surgical or cath intervention for septal reduction.
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