Cytokinetics Gains FDA Approval for MYQORZO (Aficamten) to Treat Obstructive Hypertrophic Cardiomyopathy
FDA Approves MYQORZO for Obstructive HCM
Cytokinetics, Incorporated has received U.S. Food and Drug Administration (FDA) approval for MYQORZO™ (aficamten), a cardiac myosin inhibitor, for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults. The approval is specifically aimed at improving functional capacity and symptoms for patients suffering from this chronic heart condition. This development is expected to significantly impact the therapeutic landscape for oHCM, a disease characterized by the thickening of the heart muscle, which can impede blood flow.
The approval of aficamten follows positive results from clinical trials, including the SEQUOIA-HCM study. Martin Maron, M.D., Director of the Hypertrophic Cardiomyopathy Center at Lahey Hospital and Medical Center and Principal Investigator of SEQUOIA-HCM, highlighted the significance of the approval.
“This approval of a new drug, MYQORZO, represents a meaningful addition to the treatment options available for symptomatic obstructive HCM patients,” said Dr. Maron.
Safety Restrictions and Commercial Strategy
A critical component of the approval is the requirement for a restricted distribution program. Because of the inherent risk of heart failure due to systolic dysfunction, MYQORZO is available only through a mandatory Risk Evaluation and Mitigation Strategy (REMS) program, known as the MYQORZO REMS Program. Healthcare providers must interrupt the dose of MYQORZO if a patient's Left Ventricular Ejection Fraction (LVEF) falls below 40% or if the patient experiences symptoms of heart failure or worsening clinical status due to systolic dysfunction.
Despite the safety restrictions, analysts suggest that MYQORZO's label offers subtle practical advantages over competing therapies, notably providing more flexible dosing options. This flexibility is anticipated to enhance real-world usability for physicians and patients.
Cytokinetics management has outlined ambitious commercial targets for aficamten, aiming for substantial market penetration in the coming years. Key commercial goals include:
- Achieving a greater than 50% preference share in the oHCM market.
- Increasing the overall category penetration of oHCM treatments to the mid-to-high 20% range by the end of 2026.
These targets imply a strategy of gradual adoption, supported by patient assistance programs, as the company seeks to displace or capture market share from existing treatments like CAMZYOS. The company views the approval not just as a regulatory milestone but as a catalyst for expanding the overall market for targeted HCM therapies.
Global Reach and Market Implications
The U.S. FDA approval complements an earlier regulatory success, as MYQORZO (aficamten) has also been approved by the China National Medical Products Administration for the treatment of adults with symptomatic obstructive HCM. This dual approval positions Cytokinetics to address a significant global patient population.
The entry of MYQORZO into the U.S. market intensifies competition within the cardiac myosin inhibitor class. The commercial success of aficamten will depend on its ability to demonstrate superior real-world safety and efficacy profiles, particularly concerning the management of LVEF and the flexibility afforded by its dosing regimen compared to its competitors. The focus on improving functional capacity and symptoms addresses a high unmet need within the oHCM patient community, potentially driving strong demand for the new therapy.
