Drug Trials Snapshots: MYQORZO
HOW TO USE THIS SNAPSHOT
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The "MORE INFO" bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.
LIMITATIONS OF THIS SNAPSHOT
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.
Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the MYQORZO Prescribing Information for all the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).
Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).
MYQORZO (aficamten)
(My-kor-zo)
CYTOKINETICS
Approval date: December 19, 2025
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
MYQORZO is a cardiac myosin inhibitor used for treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.
How is this drug used?
MYQORZO is a tablet that is taken once a day.
Who participated in the clinical trials?
The FDA approved MYQORZO based on evidence from a clinical trial of 282 patients with symptomatic oHCM. The trial was conducted at 82 sites in 14 countries including China, Czech Republic, Germany, Denmark, Spain, France, the United Kingdom, Hungary, Israel, Italy, Netherlands, Poland, Portugal, and the United States. The trial enrolled 94 patients from the United States and 188 patients from outside the United States. The same trial was used to assess the efficacy and safety of MYQORZO.
How were the trials designed?
MYQORZO was evaluated in SEQUOIA-HCM (NCT05186818), a randomized, double-blind, placebo-controlled trial in 282 adults with symptomatic New York Heart Association (NYHA) class II and III oHCM. Symptomatic oHCM patients were randomly assigned to receive either MYQORZO or placebo once a day. The benefit of MYQORZO was evaluated by the patient's performance on exercise testing using a bicycle or treadmill.
How were the trials designed?
MYQORZO was evaluated in SEQUOIA-HCM, a randomized, double-blind, placebo-controlled trial in 282 adults with symptomatic NYHA class II and III oHCM, left ventricular ejection fraction (LVEF) ≥60%, and resting and post-Valsalva peak left ventricular outflow track-gradient (LVOT-G) ≥30 and ≥50 mmHg at screening, respectively.
Patients were randomized in a 1:1 ratio to receive either MYQORZO or placebo once daily for 24 weeks. Patients were initiated on MYQORZO at a dose of 5 mg once daily. Doses were individually titrated at Weeks 2, 4, and 6 if the Valsalva LVOT-G was ≥30 mmHg and LVEF was ≥55%. Titration was in 5 mg dose increments up to a maximum dose of 20 mg once daily.
The primary endpoint was change from baseline in peak oxygen uptake (pVO2) at Week 24 by Cardiopulmonary Exercise Testing (CPET).
DEMOGRAPHICS SNAPSHOT
Figure 1 shows how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy and side effects of MYQORZO.
Figure 1. Baseline Demographics by Sex
Source: Adapted from FDA Review
Figure 2 shows how many patients by race were enrolled in the clinical trial used to evaluate the efficacy and side effects of MYQORZO.
Figure 2. Baseline Demographics by Race
Source: Adapted from FDA Review
Figure 3 shows how many patients by age were enrolled in the clinical trial used to evaluate the efficacy and side effects of MYQORZO.
Figure 3. Baseline Demographics by Age
Source: Adapted from FDA Review
Figure 4 shows how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy and side effects of MYQORZO.
Figure 4. Baseline Demographics by Ethnicity
Source: Adapted from FDA Review
Who participated in the trials?
Table 1. Baseline Demographics, SEQUOIA-HCM
| Demographics | MYQORZO N=142 | Placebo N=140 | Overall N=282 |
|---|---|---|---|
| Sex, n (%) | |||
| Male | 86 (60.6) | 81 (57.9) | 167 (59.2) |
| Female | 56 (39.4) | 59 (42.1) | 115 (40.8) |
| Age, years | |||
| Mean (SD) | 59 (12.6) | 59 (13.4) | 59.1 (12.94) |
| Median (min, max) | 59.0 (18, 83) | 60.0 (23, 84) | 59.5 (18, 84) |
| Age group, years, n (%) | |||
| ≥18 to <65 | 85 (59.9) | 84 (60.0) | 169 (59.9) |
| ≥65 to <75 | 45 (31.7) | 37 (26.4) | 82 (29.1) |
| ≥75 | 12 (8.4) | 19 (13.6) | 31 (11.0) |
| Race, n (%) | |||
| White | 108 (76.1) | 115 (82.1) | 223 (79.1) |
| Black or African American | 3 (2.1) | 0 | 3 (1.1) |
| Asian or Pacific Islander | 29 (20.4) | 25 (17.9) | 54 (19.1) |
| Other | 2 (1.4) | 0 | 2 (0.7) |
| Ethnicity, n (%) | |||
| Hispanic or Latino | 3 (2.1) | 6 (4.3) | 9 (3.2) |
| Not Hispanic or Latino | 128 (90.1) | 130 (92.9) | 258 (91.5) |
| Unknown | 11 (7.7) | 4 (2.9) | 15 (5.3) |
Source: Adapted from FDA Review
Abbreviations: SD, standard deviation
What are the benefits of this drug?
MYQORZO may improve your symptoms and your ability to be active.
What are the benefits of this drug (results of trials used to assess efficacy)?
In SEQUOIA-HCM, the primary efficacy endpoint of change from baseline in pVO2 to Week 24 was greater with MYQORZO compared to placebo as shown in Table 2.
Table 2. Change From Baseline in pVO2 by CPET at Week 24, SEQUOIA-HCM
| pVO2 | MYQORZO N=142 | Placebo N=140 |
|---|---|---|
| Baseline (mL/min/kg), mean (SD) | 18.4 (4.4) | 18.6 (4.5) |
| Change from baseline to Week 24 | ||
| LS mean (SE)1 | 1.7 (0.3) | 0.0 (0.3) |
| LS mean difference (95% CI)1 | 1.7 (1.0, 2.4) | |
| p-value | <0.0001 | |
Source: Adapted from MYQORZO Prescribing Information
1 LS mean (SE), and LS mean difference (95% CI) were estimated from an ANCOVA model. A placebo-based multiple imputation approach was used to impute the missing data.
Abbreviations: ANCOVA, analysis of covariance; CI, confidence interval; CPET, Cardiopulmonary Exercise Testing; LS, least square; pVO2, peak oxygen uptake; SD, standard deviation; SE, standard error
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: MYQORZO worked similarly in males and females.
- Race: The number of patients of races other than White was small; therefore, differences in how MYQORZO worked among races could not be determined.
- Age: MYQORZO worked similarly across age groups.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?
Table 3. Change From Baseline in pVO2 by CPET at Week 24 by Subgroup, Primary Efficacy Population
| Group | Subgroup | LS Mean Difference (95% CI) |
|---|---|---|
| Sex | Male | 1.7 (0.8, 2.5) |
| Female | 1.5 (0.5, 2.5) | |
| Age, years | <65 | 1.8 (1.0, 2.7) |
| ≥65 | 1.4 (0.4, 2.4) | |
| Race | Asian | 1.8 (0.5, 3.2) |
| White | 1.6 (0.9, 2.4) | |
| Ethnicity | Hispanic or Latino | 2.4 (0.03, 4.7) |
| Not Hispanic or Latino | 1.7 (1.0, 2.4) | |
| Not reported | 1.9 (-0.2, 3.4) |
Source: Adapted from FDA Review
Note: Treatment differences and credible intervals include the relevance of outcomes from other subgroups.
Abbreviations: CI, confidence interval; CPET, Cardiopulmonary Exercise Testing; LS, least squares; pVO2, peak oxygen uptake
What are the possible side effects?
MYQORZO may cause serious side effects, including heart failure where the heart cannot pump with enough force. This is a serious condition that can lead to hospitalization or death. The risk of heart failure is also increased when MYQORZO is taken with certain medicines that can lower the rate of MYQORZO's metabolism, thereby causing a buildup of the drug.
Because of the risk of heart failure, MYQORZO is only available through a restricted distribution program called the MYQORZO Risk Evaluation and Mitigation Strategy (REMS) Program.
The most common side effect of MYQORZO is high blood pressure (hypertension).
What are the possible side effects (results of trials used to assess safety)?
Hypertension (high blood pressure) was the only adverse reaction occurring in >5% of patients and more commonly on MYQORZO (8%) than on placebo (2%).
Were there any differences in side effects among sex, race and age?
Subgroup analysis for the side effect of hypertension was not conducted due to the overall small number of the events reported in the clinical trial.
Were there any differences in side effects of the clinical trials among sex, race, and age groups?
Subgroup analysis for the side effect of hypertension was not conducted due to the overall small number of the events reported in the clinical trial.
GLOSSARY
CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments.
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested.
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial.
PLACEBO: An inactive substance or "sugar pill" that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo.
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.