FDA Approval of Myqorzo for oHCM: Clinical & Regulatory Implications

The FDA has approved Myqorzo (aficamten) for adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), introducing a targeted cardiac myosin inhibitor that reduces sarcomeric contractility and left‑ventricular outflow obstruction and has been shown to improve exercise capacity.

The approval highlights flexible dosing and a monitoring framework, signaling that practices will need operational pathways to safely offer this new pharmacologic option.

Current management centers on beta‑blockers and nondihydropyridine calcium‑channel blockers for symptom control, with invasive septal reduction reserved for refractory gradients and persistent symptoms. An oral myosin inhibitor provides a noninvasive option that may improve functional capacity and potentially alter the trajectory toward septal interventions. Immediate questions include patient selection, sequencing with existing agents, and how teams will operationalize initiation and follow‑up.

Safety data described an acceptable tolerability profile with no new organ‑system signals; monitoring focuses on hemodynamic effects during titration. Across the trial report, adverse events were manageable and did not indicate additional routine drug–interaction surveillance beyond the approved labeling.

The approved REMS permits structured but relatively flexible titration and reduces some traditional monitoring burdens. Operational steps include baseline echocardiography and symptom assessment, scheduled early follow‑up with interval imaging or biomarkers at defined titration milestones, and explicit assignment of titration and documentation responsibilities to clinic staff. Synchronizing REMS requirements with clinic scheduling and staffing is the rollout priority to maintain patient safety.