FDA Approval of Cytokinetics’ Myqorzo Reflects Specialty Cardio Drug Trend

On December 19, 2025, FDA approved Myqorzo (aficamten), marking a significant milestone in cardiovascular medicine and a foundational achievement for the drug’s sponsor Cytokinetics, Incorporated (1). The drug is indicated for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve symptoms and functional capacity. The approval highlights critical developments in small-molecule innovation, regulatory compliance, and complex safety monitoring strategies (1).

What are Myqorzo’s clinical profile and mechanism of action?

Myqorzo is an allosteric and reversible inhibitor of cardiac myosin motor activity (1). In patients with oHCM, the heart muscle becomes abnormally thick, leading to hypercontractility and left ventricular outflow tract obstruction. By inhibiting myosin, Myqorzo directly addresses this underlying hypercontractility to reduce obstruction and improve the heart’s pumping function.

FDA's decision was based on results from the pivotal Phase 3 SEQUOIA-HCM clinical trial. Data published in the New England Journal of Medicine demonstrated that patients treated with Myqorzo for 24 weeks experienced a statistically significant improvement in exercise capacity, measured by an increase in peak oxygen uptake of 1.8 mL/kg/min compared with baseline, while the placebo group saw no improvement (2). The treatment effect remained consistent across subgroups, including age, sex, and the use of background beta-blocker therapy.

What regulatory or quality system significance is tied to Myqorzo’s approval?

The approval of Myqorzo underscores the importance of risk evaluation and mitigation strategies (REMS) (1). The drug’s prescribing information includes a Boxed Warning regarding the risk of heart failure due to reduced left ventricular ejection fraction.

To manage this risk, the Myqorzo REMS Program mandates several operational requirements (1):

• Certification: Both prescribers and pharmacies must be certified through enrollment in the REMS program.

• Monitoring: Patients must undergo regular echocardiogram assessments to monitor for systolic dysfunction before and during treatment.

• Controlled Distribution: Wholesalers and distributors are permitted to provide the drug only to certified pharmacies.

This level of oversight is a prime example of the intersection between patient safety and pharmaceutical quality systems.

What does Myqorzo’s approval mean for drug development and global reach?

The approval also highlights advancements in dosage form design and pharmacokinetics (1). Myqorzo was engineered for a predictable exposure response and rapid onset of action, available in 5 mg, 10 mg, 15 mg, and 20 mg tablets to allow for flexible dosing.

From a global regulatory perspective, Cytokinetics is expanding its footprint rapidly. The drug received approval in China on December 17, 2025 (3) and recently gained a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use, with a European Commission decision expected in early 2026 (4).

What does Myqorzo’s approval matter to industry professionals?

The launch of Myqorzo—expected in the US in late January 2026—serves as a comprehensive case study, touching upon API and excipient interactions, specifically noting that the drug is primarily metabolized by CYP2C9 (1). This requires careful management of drug-drug interactions, as certain inhibitors or inducers can lead to heart failure or loss of efficacy.

Furthermore, the milestone reflects a broader trend in the industry toward specialty cardiovascular biopharmaceuticals and the successful translation of muscle biology research into approved therapies (1). For professionals involved in manufacturing, process scale-up, and supply chain security, the global rollout of a first-in-class myosin inhibitor with a restricted distribution model provides valuable insights into modern drug commercialization.

References

1. Cytokinetics. Cytokinetics Announces FDA Approval of MYQORZO™ (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy to Improve Functional Capacity and Symptoms. Press Release. Dec 19, 2025.
2. Maron, S; Masri, A; Nassif, M; et al. Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2024;390:1849-1861.
3. Cytokinetics. Cytokinetics Announces NMPA Approval of MYQORZO® (aficamten) in China for Patients with Obstructive Hypertrophic Cardiomyopathy. Press Release. Dec 17, 2025.
4. Cytokinetics. Cytokinetics Announces Positive CHMP Opinion of MYQORZO® (aficamten) for the Treatment of Obstructive Hypertrophic Cardiomyopathy. Press Release. Dec 12, 2025.