FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Cytokinetics, Incorporated (Nasdaq: CYTK) announced the U.S. Food and Drug Administration (FDA) approval of  Myqorzo™ (aficamten, 5 mg, 10 mg, 15 mg, 20 mg tablets for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. Myqorzo is an allosteric and reversible inhibitor of cardiac myosin motor activity. In patients with oHCM, myosin inhibition with Myqorzo reduces cardiac contractility and left ventricular outflow tract (LVOT) obstruction.

“This is a historic moment for our company and for the patients we serve, as we fulfill our promise to translate our science into medicines that may make a meaningful difference in patients’ lives,” said Robert I. Blum, Cytokinetics’ President and Chief Executive Officer. “Our first FDA approval stands as a testament to the strength of our science and the bold, trailblazing research that has defined Cytokinetics’ leadership in muscle biology. I’m pleased that the approved label and REMS reflect the distinct characteristics of Myqorzo including a straightforward, flexible dosing regimen, no requirement for drug-drug interaction monitoring and a predictable safety profile. I am profoundly grateful for the many years of passion and persistence shown by patients with obstructive HCM, as well as healthcare professionals, advocates, partners and employees who have contributed so importantly to reaching this key milestone.”

The full U.S. Prescribing Information for Myqorzo includes a Boxed WARNING for the risk of heart failure. Myqorzo reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction. Echocardiogram assessments are required prior to and during treatment with Myqorzo to monitor for systolic dysfunction. Initiation of Myqorzo in patients LVEF <55% is not recommended. Decrease the dose of Myqorzo if LVEF <50% and ≥40%. Interrupt the dose of Myqorzo if LVEF <40% or if the patient experiences heart failure symptoms or worsening clinical status due to systolic dysfunction. Because of the risk of heart failure due to systolic dysfunction, Myqorzo is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Myqorzo REMS Program. Please see additional Important Safety Information including Boxed WARNING below.

Myqorzo is expected to be available in the U.S. in the second half of January 2026. Cytokinetics will support patients with Myqorzo & You™, a personalized program for patients prescribed Myqorzo in the U.S. to help navigate the treatment journey, provide disease and product education, and offer support with insurance benefits investigations or financial assistance for those eligible. For more information, call 833-Myqorzo (833-697-6796).

“HCM is a heart muscle disease associated with a significant symptom burden. This approval of a new drug, Myqorzo, represents a meaningful addition to the treatment options available for symptomatic obstructive HCM patients,” said Martin Maron, M.D., Director, Hypertrophic Cardiomyopathy Center, Lahey Hospital and Medical Center, and Principal Investigator of SEQUOIA-HCM. “In SEQUOIA-HCM, Myqorzo improved exercise capacity and reduced symptoms while also being well-tolerated. For these reasons, Myqorzo represents an important step forward in how we care for people living with obstructive HCM.”

https://en.wikipedia.org/wiki/Aficamten

FDA Approves Myqorzo (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy

ACC: Mavacamten Efficacious for Adolescents With Obstructive Hypertrophic Cardiomyopathy

In continuation of my update on Mavacamten

Mavacamten, the first-in-class cardiac myosin inhibitor approved for treatment of symptomatic adults with obstructive hypertrophic cardiomyopathy (HCM), is efficacious for adolescents with obstructive HCM, according to a study published online March 29 in the New England Journal of Medicine to coincide with the annual meeting of the American College of Cardiology, held from March 28 to 30 in New Orleans.

Joseph W. Rossano, M.D., from the Children's Hospital of Philadelphia, and colleagues conducted a randomized international study assessing the efficacy and safety of mavacamten in adolescents (aged 12 to <18 years) with obstructive HCM. After five weeks of screening, 44 participants were randomly assigned to either mavacamten (2.5 or 5 mg/day based on body weight) or placebo for 28 weeks.

The researchers found that participants receiving mavacamten had a substantial improvement in terms of the change in Valsalva left ventricular outflow tract (LVOT) gradient from baseline to week 28, with average decreases of 48.5 and 0.5 mmHg in the mavacamten and placebo groups, respectively. Significant improvements in favor of mavacamten were also seen in secondary end points, including change in resting LVOT gradient, maximal left ventricular wall thickness, peak oxygen consumption, and symptom measures such as fatigue and shortness of breath. Troponin and peptide levels decreased in those taking mavacamten and increased in those taking placebo.

"Beyond symptom relief, there's a signal that this may be favorably remodeling the heart, which could improve the natural history of the disease," Rossano said in a statement. "This suggests that it could be important to start children on this therapy when they’re young, before they've had many decades of ongoing injury to the heart from the obstruction."

https://en.wikipedia.org/wiki/Mavacamten