Treatment landscape | CAMZYOS UK

There is currently no treatment beyond symptomatic control or invasive therapy for obstructive HCM and the principal role of pharmacological therapy is that of symptomatic relief.^1–3 Treatment approaches vary depending on symptoms and individual risk factors, and lifestyle changes (such as limiting physical activity) may be required to adjust for the condition.^4–6

Patients with symptomatic obstructive HCM are generally offered first-line pharmacological therapy with non-vasodilating beta blockers. Non-dihydropyridine calcium channel blockers, such as verapamil and diltiazem, are recommended when beta blockers are contraindicated, ineffective or not tolerated.^3–5 Disopyramide may be used as an add-on therapy if symptoms persist after beta blocker or non-dihydropyridine calcium channel blocker monotherapy, and while disopyramide may be effective it can also be poorly tolerated, thereby limiting its usage.^3–5,7

Patients have limited pharmacological treatment options if beta blockers, non-dihydropyridine calcium channel blockers, and disopyramide are ineffective or poorly tolerated⁸

While these pharmacological therapies have been the mainstay of symptomatic obstructive HCM treatment for decades, they were not developed or intended to treat this condition, and there have been no registrational randomised trials demonstrating efficacy of these agents over placebo in obstructive HCM.⁷

Current pharmacological treatments are not disease-specific^1,2,8

While current pharmacological therapies used for treating obstructive HCM may offer symptomatic relief in some patients, their use is limited by side effects and they may not provide control of LVOT gradients and associated symptoms. In addition, beta blockers, calcium channel blockers, and disopyramide do not address the underlying pathophysiology of obstructive HCM.^1,2

Septal reduction therapies (SRTs) are complex invasive procedures that carry important risks
to patients^1,2

When current pharmacological therapies are ineffective or poorly tolerated, patients may be considered to undergo invasive surgery known as SRT (alcohol septal ablation or septal myectomy). SRT is an invasive disease-modifying intervention that may be effective in patients with drug-refractory symptoms.^1–5

SRT may lead to irreversible poor outcomes, with the success of this invasive intervention depending on the experience and skill of the centres that perform these procedures, which are not universally available. In addition, SRT carries important risks to patients inherent to invasive procedures.^1,2

Limitations with current treatments for obstructive HCM

There is a need for a licensed non-invasive treatment that goes beyond symptomatic control and addresses the underlying cause of the disease^1,2,7,8

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AHA/ACC, American Heart Association/American College of Cardiology; BB, beta blocker; CCB, calcium channel blocker; ESC, European Society of Cardiology; HCM, hypertrophic cardiomyopathy; LVEF, left ventricular ejection fraction; LVOT, left ventricular outflow tract; NYHA, New York Heart Association; QoL, quality of life; SRT, septal reduction therapy.

References

Olivotto I et al. Lancet. 2020;396(10253):759–769.
Desai N et al. Clin Ther. 2022;44(1):52–66.
NICE. Available at: https://www.nice.org.uk/guidance/ta913/resources/mavacamten-for-treating-symptomatic-obstructive-hypertrophic-cardiomyopathy-pdf-82615485457861. Date accessed: January 2024.
Arbelo E et al. Eur Heart J. 2023;44(37):3503–3626.
Ommen SR et al. Circulation. 2020;142(25):e558–e631.
Zaiser E et al. J Patient Rep Outcomes. 2020;4(1):102.
Tuohy CV et al. Eur J Heart Fail. 2020;22(2):228–240.
Stătescu C et al. Int J Mol Sci. 2021;22(13):7218.