Cytokinetics Receives Positive CHMP Opinion for MYQORZO® in Treating Obstructive Hypertrophic Cardiomyopathy

On December 12, 2025, Cytokinetics, Incorporated (NASDAQ: CYTK) announced a significant development in the treatment of obstructive hypertrophic cardiomyopathy (oHCM). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending marketing authorization for MYQORZO® (aficamten). This decision paves the way for adult patients suffering from symptomatic oHCM in the European Union (EU). A final decision is expected from the European Commission in the first quarter of 2026.

Details of the Positive Recommendation

The favorable CHMP opinion is rooted in the robust clinical data generated from the SEQUOIA-HCM trial, which demonstrated the safety and efficacy of MYQORZO in treating oHCM. Robert I. Blum, President and CEO of Cytokinetics, expressed his satisfaction with the CHMP's recommendation: "We are accelerating our commercial readiness activities given the urgent need for new treatment options for the European oHCM patient community."

Insights from Clinical Trials

The pivotal Phase 3 trial, SEQUOIA-HCM, published in the New England Journal of Medicine, showcased impactful results. Key findings from the study include:

• A 24-week treatment period with MYQORZO significantly enhanced exercise capacity.
• Patients exhibited an increase in peak oxygen uptake (pVO2) of 1.8 ml/kg/min compared to no change in the placebo group.
• The treatment effect was consistent across various demographics, including age and sex.
• MYQORZO was well-tolerated, with serious adverse events reported in 5.6% of patients versus 9.3% in placebo.

MYQORZO® (Aficamten) Explained

MYQORZO® (aficamten) is a selective, small molecule cardiac myosin inhibitor aimed at reducing myocardial hypercontractility associated with HCM. Its design focuses on lowering the number of active actin-myosin cross bridges during cardiac cycles, thus providing a therapeutic option for symptomatic oHCM patients.

Ongoing Regulatory Reviews and Future Trials

MYQORZO is also under U.S. regulatory review, with the FDA examining a New Drug Application (NDA) that has a target decision date of December 26, 2025. In China, the National Medical Products Administration (NMPA) is prioritizing the review of MYQORZO as well.

Additionally, Cytokinetics is advancing further trials including ACACIA-HCM for non-obstructive HCM and CEDAR-HCM focusing on pediatric populations. These initiatives aim to evaluate MYQORZO's full therapeutic potential.

Understanding Hypertrophic Cardiomyopathy (HCM)

Hypertrophic cardiomyopathy (HCM) is a prevalent inherited cardiovascular disorder affecting approximately 1 in 500 Europeans. The condition causes abnormal thickening of the heart muscle, leading to reduced heart function and various symptoms such as chest pain and shortness of breath. Notably, two-thirds of HCM patients experience obstructive HCM (oHCM), where the thick heart muscle obstructs blood flow.

Risks Associated with HCM

Individuals diagnosed with HCM face heightened risks of cardiovascular complications, including atrial fibrillation, strokes, and sudden cardiac death—making effective treatment paramount.

About Cytokinetics

Cytokinetics has dedicated over 25 years to pioneering advancements in muscle biology and is now preparing for the potential commercialization of aficamten (MYQORZO). The company is actively pursuing regulatory approvals and has additional investigational drugs targeting heart failure and muscular dystrophies.

Conclusion

The positive CHMP opinion reflects a promising advancement in treatment options for patients with obstructive hypertrophic cardiomyopathy. As Cytokinetics continues to prepare for commercial readiness, there is hope for improved patient outcomes within the European oHCM community.