NICE Backed Aficamten the Same Day the MHRA Authorised It, at a Price It Will Not Disclose

NICE Backed Aficamten the Same Day the MHRA Authorised It, at a Price It Will Not Disclose

Athithi Verma· 31 July 2026· 2 min read· Synopulse
  • The MHRA granted MYQORZO (aficamten) a marketing authorisation across the United Kingdom for symptomatic NYHA class II-III obstructive hypertrophic cardiomyopathy in eligible adults. NICE issued positive guidance the same day, covering England and Wales.
  • The NICE recommendation is fenced. Aficamten is an option only as an add-on to individually optimised standard care, meaning beta-blockers, non-dihydropyridine calcium-channel blockers or disopyramide, or alone where those are contraindicated, and only if Cytokinetics supplies it according to the commercial arrangement.
  • The assessment drew on Phase 3 SEQUOIA-HCM: 282 adults, 142 on aficamten and 140 on placebo, over 24 weeks. Peak oxygen uptake improved by 1.8 mL/kg/min against 0.0 on placebo, a least squares mean difference of 1.7 (95% CI 1.0 to 2.4, p<0.001).
  • Most common adverse reactions were hypertension 7.7%, palpitations 7.0%, dizziness 4.2% and systolic dysfunction 3.5%. HCM affects roughly 1 in 500 Europeans and about half of those have the obstructive form. Cytokinetics expects NHS availability later this year, and the product carries the MHRA black triangle for additional monitoring.
Access read Authorisation and reimbursement almost never land on the same day. The normal sequence is a regulator first, then a NICE appraisal running for months while clinicians know a medicine exists and cannot routinely prescribe it on the NHS. Cytokinetics closed that gap to zero, and its own European head credits the collaboration that made concurrent decisions possible. That is the achievement, and it is a template worth copying. Then read the conditions, because that is where the guidance actually lives. Aficamten is recommended as an add-on to optimised standard care rather than instead of it, so patients pass through beta-blockers, calcium-channel blockers or disopyramide first, and monotherapy is reserved for those who cannot take them. Then the clause that matters most: the recommendation holds only if the company supplies the drug under the agreed commercial arrangement. NICE said yes at a price it is not publishing, which means the list price was not the price. A positive appraisal bought with a confidential discount is still a positive appraisal, but it tells you the value case needed help. Two more things to log. The authorisation covers the United Kingdom while the guidance covers England and Wales, so Scotland runs its own process and the access map is uneven on day one. And what NICE accepted here was an exercise-capacity endpoint, 1.7 mL/kg/min of peak oxygen uptake, not an events benefit. That distinction will matter when outcomes data arrives.

Read the original source (Cytokinetics) →