Cytokinetics’ Myqorzo Wins FDA Approval for oHCM, Set to Challenge BMS’s Camzyos

Cytokinetics announced the FDA approval of its oral cardiac myosin inhibitor Mycorzo (aficamten), right before Christmas, for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms.

The milestone marks Cytokinetics’ first FDA-approved medicine.

HCM is the most common inherited cardiovascular disease caused by a single gene. In the US, more than 300,000 patients are diagnosed with the disease, and Cytokinetics estimates that an additional 400,000 to 800,000 individuals remain undiagnosed.

Roughly half of HCM patients have obstructive disease (oHCM), while the other half have non-obstructive HCM (nHCM). Cytokinetics is currently conducting a Phase III trial in nHCM, where the heart muscle is thickened but does not significantly obstruct blood flow.

Myqorzo comes in 5 mg, 10 mg, 15 mg and 20 mg oral tablet strengths and works by blocking the protein myosin to reduce cardiac hypercontractility and left ventricular outflow tract (LVOT) obstruction, which are central to oHCM’s pathophysiology.

The approval was based on positive data from the pivotal SEQUOIA-HCM Phase III trial, in which patients receiving Myqorzo for 24 weeks experienced significant improvements in exercise capacity compared with placebo. For example, treatment led to a meaningful increase in peak oxygen uptake (pVO₂), a key functional measure in symptomatic oHCM.

Results from the study were published in The New England Journal of Medicine.

“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 CEO, in the company announcement.

“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.”

Martin Maron, MD, director of the Hypertrophic Cardiomyopathy Center at Lahey Hospital and a principal SEQUOIA-HCM investigator, noted that the approval represents an important step forward in care for people living with oHCM.

“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 Dr. Maron.

Patient groups also emphasized the long-awaited addition of a targeted therapy after years of limited options.

“Living with symptomatic obstructive HCM means managing physical limitations and burdensome symptoms every day of your life,” said Lisa Salberg, founder and CEO of the Hypertrophic Cardiomyopathy Association (HCMA). “For far too long, we’ve had few options to address our needs, and the approval of Myqorzo is a long-awaited and major addition to bring new hope to patients living with oHCM. We are so grateful to the team at Cytokinetics for listening to the patient community and working in true partnership to bring this therapy to so many in need.”

Myqorzo’s US label includes a boxed warning for the risk of heart failure due to reduced left ventricular ejection fraction (LVEF), as the drug works by reducing heart muscle contractions.

Because of the risk, Myqorzo is available only through a Risk Evaluation and Mitigation Strategy (REMS) program, requiring certified prescribers, pharmacies and monitoring of heart function with echocardiograms before and during treatment.

While the therapy was generally well tolerated in trials, hypertension was the most common adverse event occurring more frequently with Myqorzo versus placebo. Ongoing cardiac monitoring and careful dose adjustments based on changes in LVEF are part of safe clinical use.

Related: Tempus Scores FDA Clearance for AI ECG Software to Flag Low LVEF

In addition to US approval, Myqorzo has also been authorized in China by the National Medical Products Administration (NMPA) for adults with NYHA Class II-III oHCM, with approval triggering milestone payments under Cytokinetics’ collaboration with Sanofi.

In Europe, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization, with a final European Commission decision expected in early 2026 that could broaden patients’ access internationally.

Cytokinetics is stepping into a competitive landscape defined in part by its own past work. The company collaborated with MyoKardia in 2012 on the program that led to Bristol Myers Squibb’s (BMS) leading, pioneering oHCM drug Camzyos (mavacamten). BMS obtained it through its $13.1 billion acquisition of MyoKardia in 2020, and received FDA approval for it in 2022.

Like Myqorzo, Camzyos is also a cardiac myosin inhibitor, and both drugs are expected to reach blockbuster status. Camzyos earned $602 million in global sales in 2024 and $715 million in the first three quarters of 2025.

While Camzyos also carries a boxed warning and REMS restrictions, analysts say the two drugs have significant clinical and safety differentiators.

According to reporting by Fierce Pharma, Mizuho Securities’ Salim Syed notes that “Myqorzo seems the safer drug and easier to use,” which is particularly key for treatment-naïve patients, he said.

Evercore ISI’s Cory Kasimov said the drugs are “clearly differentiated,” citing Myqorzo’s unique kinetics and profile, which he expects will appeal to high-prescribing physicians and support its use as a first-line option in new oHCM patients.

Blum also noted that he was, “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.”

Cytokinetics said it plans to launch Myqorzo in the second half of January.

The company has not disclosed its pricing, but it is expected to be around Camzyos’ annual wholesale acquisition cost of around $104,000.