Aficamten approved in the EU for symptomatic obstructive HCM
The cardiac myosin inhibitor aficamten has been approved by the European Commission (EC) for the treatment of adults with symptomatic (NYHA class II-III) obstructive HCM.
The EC has approved aficamten (5 mg, 10 mg, 15 mg and 20 mg tablets) for the treatment of adults with symptomatic (NYHA class II-III) obstructive HCM. This approval was based on positive results of the phase 3 SEQUOIA-HCM trial.
SEQUOIA-HCM was a multicenter, randomized, placebo-controlled, double-blind trial in which 282 patients with symptomatic obstructive HCM were randomized to aficamten or placebo for 24 weeks. The primary endpoint was change in peak oxygen uptake (pVO2) as measured with cardiopulmonary exercise testing from baseline to week 24. In SEQUOIA-HCM, treatment with aficamten significantly improved pVO2 in patients with obstructive HCM compared with placebo (least square mean difference: 1.74 mL/kg/min; 95%CI: 1.04-2.44; P=0.000002). The treatment effect of aficamten was consistent across all prespecified subgroups.
The most commonly reported adverse reactions of aficamten in SEQUOIA-HCM included hypertension (7.7%), palpitations (7%), dizziness (4.2%), and systolic dysfunction defined as LVEF <50% (3.5%).
Aficamten is an allosteric, reversible inhibitor of cardiac myosin and reduces LVOT obstruction by lowering cardiac contractility.