MHRA grants marketing authorisation in UK for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in eligible adult patients – Cytokinetics

Cytokinetics, Incorporated announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted MYQORZO (aficamten) a marketing authorisation across the United Kingdom for the treatment of symptomatic (New York Heart Association, NYHA class II-III) obstructive hypertrophic cardiomyopathy (oHCM) in eligible adult patients. At the same time the National Institute for Health and Care Excellence (NICE) has issued guidance recommending aficamten for use in eligible adult patients in England and Wales.

The guidance from NICE states that aficamten can be used as an option to treat symptomatic oHCM in eligible adults with a NYHA class of II to III. It can only be used as an add-on to individually optimised standard care including beta-blockers, non-dihydropyridine calcium-channel blockers or disopyramide, or alone if these treatments are contraindicated, and if the company provides it according to the commercial arrangement.

“We are pleased to have secured a marketing authorisation for aficamten and to see positive guidance from NICE for eligible adult patients in England and Wales,” said Joseph Dagher, Senior Vice President and Head of Europe, Cytokinetics. “We are grateful for the collaborations that made this review possible for concurrent MHRA and NICE decisions and are committed to supporting access for eligible adult patients through the NHS upon availability expected later this year.”

The MHRA assessment of aficamten drew on data from SEQUOIA-HCM, a phase 3 multicentre double-blind placebo-controlled randomised study in 282 adults (142 aficamten, 140 placebo) treated for 24 weeks. The primary endpoint was the change from baseline to Week 24 in peak oxygen uptake (pVO₂) measured by cardiopulmonary exercise testing. Aficamten resulted in a statistically significant improvement in peak oxygen uptake compared with placebo (mean change 1.8 mL/kg/min with aficamten versus 0.0 mL/kg/min with placebo; least squares mean difference 1.7 mL/kg/min, 95% confidence interval 1.0 to 2.4; p < 0.001).

The most commonly reported adverse reactions with aficamten were hypertension (7.7%), palpitations (7.0%), dizziness (4.2%) and systolic dysfunction (3.5%).

MYQORZO (aficamten) is subject to additional monitoring. This will allow quick identification of new safety information.