Camzyos: A High-Conviction Growth Play in Rare Disease Innovation

Bristol Myers Squibb’s Camzyos (mavacamten) has emerged as a transformative therapy for obstructive hypertrophic cardiomyopathy (oHCM), a rare but debilitating cardiovascular disease. With real-world evidence reinforcing its clinical value and global adoption expanding rapidly, Camzyos is reshaping the competitive landscape in rare disease innovation. For investors, the drug represents a high-conviction growth play, underpinned by a durable competitive moat built on clinical differentiation, regulatory leadership, and strategic market access.

Clinical Differentiation: Real-World Evidence Validates Efficacy

Camzyos’s success hinges on its ability to deliver measurable improvements in patient outcomes. Real-world data from the global WAYFARER-HCM program, including the COLLIGO-HCM study, demonstrate that 59.9% of patients achieved a ≥1 NYHA class improvement by week 24, with 86.5% achieving NYHA class II or below by week 96 [1]. These results mirror clinical trial outcomes, such as the Phase III EXPLORER-HCM trial, where 65% of patients on Camzyos improved by one or more NYHA classes compared to 31% on placebo [6]. Crucially, the drug maintains cardiac function, with mean left ventricular ejection fraction (LVEF) remaining above 61% in real-world studies [1].

The FDA’s recent label updates further strengthen Camzyos’s appeal. By reducing the frequency of required echocardiography monitoring and removing contraindications with common drug interactions, the revised prescribing information simplifies treatment and expands eligibility [3]. These changes address prior limitations, such as the need for frequent cardiac monitoring, which had constrained adoption [4].

Global Adoption and Market Access: A Strategic Moat

Camzyos has secured regulatory approvals in over 50 countries, including the U.S. and EU, where it is the first and only cardiac myosin inhibitor approved for oHCM [1]. Its inclusion in both ESC and AHA/ACC guidelines solidifies its position as a standard of care [1]. However, market access remains a challenge. The drug’s annual list price of $110,000 in the U.S. raises affordability concerns, though Bristol Myers Squibb has launched patient support programs to mitigate this [2].

Reimbursement hurdles persist in Asia, particularly in South Korea, where negotiations with the National Health Insurance Service (NHIS) have stalled [6]. This highlights the need for continued advocacy and real-world evidence generation to secure broader coverage. Despite these challenges, revenue projections are optimistic, with Camzyos expected to reach $500 million annually by 2026 [2].

Competitive Landscape: Navigating Rivals and Unmet Needs

Camzyos faces competition from Cytokinetics’ aficamten, another cardiac myosin inhibitor. While aficamten showed a 42% composite endpoint improvement in Phase 3 trials (versus 37% for Camzyos), its lower incidence of LVEF dropping below 50% (3.5% vs. 6%) may appeal to payers and clinicians [2]. However, Camzyos’s first-mover advantage and robust real-world evidence base provide a significant edge.

The drug’s failure in the Phase III ODYSSEY-HCM trial for non-obstructive HCM (nHCM) underscores the importance of disease-specific targeting [5]. While this limits its applicability, it also reinforces the need for therapies like Camzyos in oHCM, where no alternatives exist.

Conclusion: A Durable Growth Engine

Camzyos exemplifies Bristol Myers Squibb’s ability to innovate in rare diseases, combining clinical excellence with strategic market access. While challenges in nHCM and reimbursement remain, the drug’s entrenched role in oHCM and expanding real-world evidence position it as a long-term growth driver. For investors, Camzyos represents more than a product—it’s a testament to the power of precision medicine in addressing unmet medical needs.

Source:
[1] Bristol Myers Squibb Presents Real-World Outcomes of Camzyos (mavacamten) Across Four Continents at the European Society of Cardiology (ESC) Congress 2025 [https://news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Presents-Real-World-Outcomes-of-Camzyos-mavacamten-Across-Four-Continents-at-the-European-Society-of-Cardiology-ESC-Congress-2025/default.aspx]
[2] Cardiomyopathy Market Predicted to Grow at a Steady CAGR of 6.13% during 2025-2035, Impelled by Rising Prevalence of Cardiovascular Diseases [https://www.biospace.com/press-releases/cardiomyopathy-market-predicted-to-grow-at-a-steady-cagr-of-6-13-during-2025-2035-impelled-by-rising-prevalence-of-cardiovascular-diseases]
[3] U.S. Food and Drug Administration Updates CAMZYOS® (mavacamten) Label to Reduce Echocardiography Monitoring Requirements and Contraindications [https://investors.bms.com/iframes/press-releases/press-release-details/2025/U-S--Food-and-Drug-Administration-Updates-CAMZYOS-mavacamten-Label-to-Reduce-Echocardiography-Monitoring-Requirements-and-Contraindications/default.aspx]
[4] FDA Updates Prescribing Information for Bristol Myers ... [https://www.pharmexec.com/view/fda-updates-prescribing-information-bristol-myers-squibb-camzyos-reduce-echocardiography-monitoring-requirements]
[5] Bristol Myers Squibb's Camzyos Falls Short in Phase 3 ... [https://www.biospace.com/press-releases/bristol-myers-squibb-provides-update-on-phase-3-odyssey-hcm-trial]
[6] CAMZYOS® (mavacamten) oHCM Clinical Study Results [https://www.camzyos.com/what-is-camzyos/study-results]