The medical treatment arsenal for HCM is expanding

While beta-blockers remain the first-line therapy for patients with obstructive hypertrophic cardiomyopathy (HCM), additional treatment strategies are available and continue to be refined. In contrast, therapeutic options for non-obstructive HCM  remain limited, and the unmet clinical need is even greater. Several established strategies continue to be evaluated to optimise outcomes in this population.

Dr Caroline Coats (University of Glasgow, UK) discussed current treatment options in HCM [1]. “The symptoms in HCM can be driven by various underlying mechanisms, including cardiac hypertrophy, left ventricular outflow tract (LVOT) obstruction, myocardial ischaemia, small vessel disease, diastolic dysfunction, and pulmonary hypertension,” explained Dr Coats. “Therefore, there are many treatment targets in HCM.” Beta-blockers are currently the first-line therapy for patients with obstructive HCM [2]. If a patient remains symptomatic or is intolerant to beta-blockers, they can switch to calcium channel inhibitors. Patients who do not respond to first-line pharmacological therapy may require escalation to interventional approaches. “If a patient is still symptomatic after these options have failed, the treating physician may consider interventional options, like septal reduction therapy,” said Dr Coats.

She further outlined that only a few randomised-controlled trials have been performed in HCM. Concerning beta-blockers, a recent trial displayed that these agents lower LVOT gradient and reduce exertional dyspnoea and angina, but do not improve exercise capacity [3]. “At the sarcomere level, HCM is primarily a disease of hypercontractility,” continued Dr Coats. “Mavacamten is a first-in-class cardiac myosin inhibitor that is designed to reduce the excess actin-myosin cross-bridging and myosin ATPase activity.” This agent met its primary endpoints of pVO₂ and NYHA in the EXPLORER-HCM trial (NCT03470545) and strongly reduced the need for septum reduction therapy in the VALOR-HCM study [4,5]. In non-obstructive HCM, efficacious pharmacological options are limited. “Recently, the large ODYSSEY-HCM trial delivered neutral results, with mavacamten not improving pVO₂ and KCCQ-CSS compared with placebo in patients with non-obstructive HCM (n=590),” mentioned Dr Coats. Nonetheless, the search for effective treatment strategies in this population remains a priority, with ongoing research focused on optimising current approaches and improving outcomes for patients with non-obstructive HCM.

In conclusion, the treatment armamentarium for HCM continues to evolve, with established therapies forming the foundation of care for obstructive forms of the disease. While novel treatment options are under investigation, current management remains focused on symptom control and haemodynamic improvement. Ongoing research is aimed at optimising existing strategies and addressing the persistent unmet need in non-obstructive HCM.

1. Coats C. Medical therapy for hypertrophic cardiomyopathy. Hypertrophic cardiomyopathy: diagnosis and management. Heart Failure 2025, 19 May, Belgrade, Serbia
2. Arbelo E, et al. Eur Heart J. 2023;44(37):3503-3623.
3. Dybro AM, et al. J Am Coll Cardiol. 2021;78(25):2505-2517.
4. Olivotto I, et al. Lancet. 2020;396(10253):759-769.
5. Desai MY, et al. J Am Coll Cardiol. 2022;80(2):95-108.

Medical writing support was provided by Dr Rachel Giles.

 

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Posted on 24 June 202526 June 2025