FDA approves aficamten-MYQORZO for the treatment of obstructive hypertrophic cardiomyopathy

On December 19, 2025, Cytokinetics announced that the drug it developed, aficamten (aficamten)-MYQORZO, had obtained U.S. Food and Drug Administration approval. Approved by the Food and Drug Administration (FDA) for the treatment of adult patients with symptomatic obstructivehypertrophic cardiomyopathy (oHCM), with the goal of improving the patient's functional abilities and symptoms. Afkateis an allosteric and reversible inhibitor of cardiac myosin motor activity, which relieves left ventricular outflow tract (LVOT) obstruction by inhibiting myocardial contractility.

1. The treatment principle of Afkatai

The mechanism of action of Afkatai is to reduce myocardial contractility by inhibiting cardiac myosin activity, thereby reducing left ventricular outflow tract obstruction. This mechanism is particularly important in patients with obstructive hypertrophic cardiomyopathy, since heart obstruction in this condition is often caused by excessive contraction of the heart muscle. By inhibiting this excessive contraction, Afkate can significantly improve patients' symptoms and mobility.

2. FDA approval background and significance

The FDA's approval is based on Afkat's excellent performance in clinical trials, especially the positive results in the SEQUOIA-HCM pivotal Phase 3 clinical trial. This approval makes Afuketai a new drug option for the treatment of obstructive hypertrophic cardiomyopathy, filling the treatment gap in this field and providing patients with more treatment options.

3. Afkatai’s clinical trial results

In the SEQUOIA-HCM trial, the use of Afkatide improved patients' exercise capacity and symptoms, especially their maximum oxygen uptake (pVO2) during the cardiopulmonary exercise test (CPET). The specific results are as follows:

1. Maximum oxygen uptake increase: The pVO2 of patients treated with Afkatide increased by 1.8 ml/kg/min within 24 weeks, while patients using placebo did not increase. The p value was 0.000002, indicating that Afkatet has a significant effect in improving patients' exercise capacity.

2. Improvement of cardiac function: In patients using Afkatai, the left ventricular ejection fraction (LVEF) detected by echocardiography improved. Although some patients had a decrease in LVEF, the overall tolerance was good.

3. In addition, Afkatide demonstrated consistent therapeutic effects across all patient subgroups, regardless of patient age, gender, or background beta-blocker therapy.

4. The safety of Afkaita

In clinical trials, the safety profile of Afkatide was favorable, and there were no cases of worsening of heart failure or treatment interruption due to lowlow LVEF. Compared with the placebo group, the incidence of serious adverse reactions in the Afkatide group was lower, 5.6% and 9.3% respectively. Here are some key adverse reactions:

1. Hypertension: The proportion of hypertension in the Afkatide group was 8%, which was significantly higher than the 2% in the placebo group. This increase in blood pressure is consistent with resolution of LVOT obstruction and improvement in cardiac output.

2. Decreased LVEF: 5 patients (3.5%) using Afkatide had LVEF less than 50%, compared with 0.7% in the placebo group.

Despite the occurrence of adverse reactions such as hypertension, Afkatide was generally well tolerated, and no major safety issues arose during treatment.

5. Dosing regimen of Afkatai

1. The recommended dose of Afkatai is: Initial dose: 5 mg on day 1 of cycle 1;

2. Subsequent doses: 10 mg on days 8 and 15, and then every two weeks until disease progression or unacceptable toxicity occurs.

3. The administration method of Afkatai is simple and flexible, and patients can use it under the guidance of doctors to better control the condition and reduce side effects.

6. The Prospects and Outlook of Afkaita

The approval of Afkatib brings new hope to patients with obstructive hypertrophic cardiomyopathy. Obstructive hypertrophic cardiomyopathy is a common hereditary heart disease that causes limited cardiac function and reduced quality of life in patients. Afkatide significantly improves patients' exercise capacity and quality of life by improving cardiac contractility and relieving obstruction. With the accumulation of more clinical data, Afkatai is expected to become a new standard in the treatment of obstructive hypertrophic cardiomyopathy and provide patients with better treatment options.

7. The future development of Afkaitai

The launch of Afkatai not only provides new treatment options foroHCM patients, but may also play an important role in the treatment of other heart diseases and diseases. With the further clinical application of this drug, its efficacy and safety will continue to be verified and benefit more patients.

Keyword tag:FDA approved, aficamten, obstructive hypertrophic cardiomyopathy, oHCM, Sequoia-HCM, heart disease, exercise capacity, heart failure, elevated blood pressure

References: Updated on December 19, 2025,https://www.drugs.com/newdrugs/fda-approves-myqorzo-aficamten-adults-symptomatic-obstructive-hypertrophic-cardiomyopathy-6699.html