Aficamten

Aficamten is an allosteric and reversible inhibitor of cardiac myosin motor activity. Aficamten reduces the force generated by myosin at the cardiac sarcomere, which contributes to the pathophysiology of HCM.

• MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. MYQORZO is a cardiac myosin inhibitor indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.

• MYQORZO is contraindicated with concomitant use of rifampin [see Warnings and Precautions (5.3) and Drug Interactions (7.1) ] .contraindicated

Recommended starting dose is 5 mg orally once daily. ( 2.1 ) Dosage is individualized based on echocardiographic assessments and clinical status. Refer to Full Prescribing Information for instructions on dosage modification. ( 2.1 , 2.2 ) 2.1 Evaluation Before and During Use of MYQORZO Initiation or up-titration of MYQORZO in patients with LVEF <55% is not recommended. Patients may develop heart failure while taking MYQORZO. Regular LVEF and Valsalva left ventricular outflow tract gradient (LVOT-G) assessment is needed for titration to achieve an appropriate target Valsalva LVOT-G, while maintaining LVEF ≥50% and avoiding heart failure symptoms. 2.2 Recommended Dosage and Administration The recommended starting dose of MYQORZO is 5 mg orally once daily. Increase the dose every 2 to 8 weeks by 5 mg until a maintenance dose or the maximum recommended dose of 20 mg once daily is achieved. The maintenance dose of MYQORZO is individualized based on the patient's LVEF and LVOT-G. Recommendations for dosing based on LVEF and LVOT-G criteria are provided in Table 1.

Risk of Heart Failure : Consider decreasing dose or dose interruption in patients with serious intercurrent illness. ( 2.2 , 5.1 ) Drug Interactions Leading to Increased Risk of Heart Failure or Loss of Effectiveness: Advise patients of the potential for drug interactions. ( 2.3 , 5.3 , 17 ) 5.1 Heart Failure MYQORZO reduces cardiac contractility, which can reduce LVEF and cause heart failure. Patients who experience a serious intercurrent illness (e.g., serious infection) or arrhythmia (e.g., new or uncontrolled atrial fibrillation) may be at greater risk of developing systolic dysfunction and heart failure [see Clinical Trial Experience (6.1) ] . Asymptomatic LVEF reduction, intercurrent illnesses, and arrhythmias require additional monitoring considerations [see Dosage and Administration (2.2) ]. Assess the patient's clinical status and LVEF prior to and regularly during treatment and adjust the MYQORZO dose accordingly [see Dosage and Administration (2.3) ]. New or worsening arrhythmia, dyspnea, chest pain, fatigue, leg edema, or elevations in N-terminal pro-B-type natriuretic peptide (NT-proBNP) may be signs and symptoms of heart failure and should prompt an evaluation of cardiac function. Initiation of MYQORZO in patients with LVEF <55% is not recommended. 5.2 MYQORZO REMS Program MYQORZO is available only through a restricted program called the MYQORZO REMS Program, because of the risk of heart failure due to systolic dysfunction [see Warnings and Precautions (5.1) ].

The following adverse reaction is discussed in other sections of labeling: Heart Failure [see Warnings and Precautions (5.1) ] The most common adverse reaction was hypertension (8%). ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Cytokinetics at 1-833-633-2986 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety of MYQORZO was evaluated in SEQUOIA-HCM, a phase 3, randomized, double-blind, placebo-controlled study [see Clinical Studies (14) ] . Of the 282 adults with oHCM, 142 patients received daily doses of MYQORZO (initiated at 5 mg and titrated up to a maximum dose of 20 mg) and 140 patients received placebo. The median treatment duration for patients receiving MYQORZO was ~24 weeks (range 4 to 29 weeks). Hypertension (8% vs. 2%) was the only adverse reaction occurring in >5% of patients and more commonly on MYQORZO than on placebo. Eligible oHCM patients were able to participate in an ongoing, open-label, single-arm, long-term safety study (FOREST-HCM). Based on available data, the safety profile of MYQORZO in FOREST-HCM was similar to that observed in SEQUOIA-HCM. Effects on Systolic Function In SEQUOIA-HCM, the mean (SD) resting LVEF at baseline was 75% (6) in both treatment groups.

8.1 Pregnancy Risk Summary There are no available data on the use of MYQORZO during pregnancy to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. The underlying maternal condition during pregnancy poses a risk to the mother and fetus (see Clinical Considerations ) . In embryo-fetal and pre- and postnatal development studies, when pregnant rats were administered aficamten during the period of organogenesis, aficamten increased the incidence of structural malformations at exposures ≥4-times the maximum recommended human dose (MRHD) of 20 mg based on free area under the concentration curve (AUC) . No adverse effects on development were seen at 3-times the MRHD exposure (see Data ) . The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defects, loss, and other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. There is a pregnancy safety study for MYQORZO.

Metabolism

Aficamten exposure increases dose proportionally following single doses of 1 mg to 75 mg and multiple once daily doses of 5 mg to 20 mg. Aficamten pharmacokinetics was comparable between healthy subjects and patients with oHCM.

Clinical Experience and Effects There have been no reports of overdose with MYQORZO. Cardiovascular effects of overdose may include reduced LVEF, heart failure, and hypotension. Signs and symptoms of heart failure such as dyspnea, fatigue, leg edema, or elevations in NT-proBNP should prompt an evaluation of cardiac function [see Warnings and Precautions (5.1) ]. Management Discontinue MYQORZO treatment. Provide medically supportive measures to maintain hemodynamic stability and monitor left ventricular function. Consider contacting the Poison Help line (1-800-222-1222) or a medical toxicologist for additional overdose management recommendations.

• Dec 19, 2025NDANDA219083Cytokinetics

1. 1Dyspnoea240%
2. 2Atrial Fibrillation120%
3. 3Blood Pressure Increased120%
4. 4Chest Pain120%
5. 5Headache120%
6. 6Myositis120%
7. 7Nausea120%
8. 8Presyncope120%
9. 9Product Dose Omission Issue120%
10. 10Pulmonary Hypertension120%
11. 11Rhabdomyolysis120%

The 10 most recently updated of 12 ClinicalTrials.gov registrations naming Aficamten as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Metoprolol Succinate in Adults With Symptomatic oHCM
  Completed · Phase 3 · Interventional · 175 enrolled · Cytokinetics
  NCT05767346updated 2026-06-09
• A Study to Learn More About How Well Aficamten Works in Japanese Participants With Symptomatic Obstructive Hypertrophic Cardiomyopathy
  Active not recruiting · Phase 3 · Interventional · 36 enrolled · Bayer
  NCT07023341updated 2026-06-02
• Mavacamten to Aficamten Transition in Patients With Obstructive Hypertrophic Cardiomyopathy
  Recruiting · Phase 4 · Interventional · 40 enrolled · Oregon Health and Science University
  NCT07600177updated 2026-05-20
• A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
  Recruiting · Phase 2 · Phase 3 · Interventional · 55 enrolled · Cytokinetics
  NCT06412666updated 2026-04-13
• Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM
  Completed · Phase 3 · Interventional · 282 enrolled · Cytokinetics
  NCT05186818updated 2026-03-03
• Dose-finding Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CK-3773274 in Adults With Hypertrophic Cardiomyopathy
  Completed · Phase 2 · Interventional · 96 enrolled · Cytokinetics
  NCT04219826updated 2026-02-24
• Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM
  Active not recruiting · Phase 3 · Interventional · 500 enrolled · Cytokinetics
  NCT06081894updated 2026-02-02
• Open-label Extension Study to Evaluate the Long-term Safety and Tolerability of Aficamten in Adults With HCM
  Enrolling by invitation · Phase 2 · Phase 3 · Interventional · 900 enrolled · Cytokinetics
  NCT04848506updated 2026-01-22
• An Open-Label Study of Aficamten for Chinese Patients With Symptomatic oHCM
  Completed · Phase 3 · Interventional · 40 enrolled · Corxel Pharmaceuticals
  NCT06116968updated 2026-01-21
• Safety, Tolerability and Pharmacokinetics Study of CK-3773274
  Completed · Phase 1 · Interventional · 28 enrolled · Corxel Pharmaceuticals
  NCT04783766updated 2025-01-24

Frequently asked questions

How does Aficamten work?

Aficamten is an allosteric and reversible inhibitor of cardiac myosin motor activity. Aficamten reduces the force generated by myosin at the cardiac sarcomere, which contributes to the pathophysiology of HCM.

What is Aficamten used for?

According to FDA labeling, Aficamten carries indications including: MYQORZO is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms. MYQORZO is a cardiac myosin inhibitor indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Aficamten?

Aficamten is classified as Cardiac Myosin Inhibitors, Cardiac Contractility Alteration, Negative Inotropy.

What are the brand names for Aficamten?

Aficamten is marketed under brand names including Myqorzo.

What are the contraindications for Aficamten?

Aficamten labeling lists contraindications including: MYQORZO is contraindicated with concomitant use of rifampin [see Warnings and Precautions (5.3) and Drug Interactions (7.1) ] .. Always consult the full prescribing information and a clinician.