Spherix Global Insights’ new patient audit on transfusion-dependent thalassemia reveals strong physician interest in emerging treatments like mitapivat and etavopivat, and provides insight into ideal candidates for curative gene therapies.
[Exton, PA, June 04, 2025] – Despite the promise of new curative strategies for beta thalassemia, such as Casgevy (Vertex) and Zynteglo (bluebird bio), most transfusion-dependent thalassemia patients remain tethered to long-term transfusion support. Transfusions, while providing symptom relief, in turn create logistical barriers and their own clinical challenges. New data from Spherix Global Insights’ independent chart audit, Patient Chart Dynamix™: Transfusion Dependent Thalassemia 2025 (US), is based on insight from 81 real-world patient charts submitted by 49 US-based hematologists. These data paint a complex picture of therapeutic inertia, growing anticipation for emerging treatments, and deep-seated frustration with limitations of current treatment options.
Most transfusion-dependent beta thalassemia patients follow some regular transfusion schedule. That said, many receive them at relatively extended intervals: often once a month or even less frequently, though a small subset still require more frequent transfusions. This variation in treatment patterns, along with persistent symptoms and unmet needs highlighted in the chart audit, points to a strong opportunity for new therapies to improve care for a broad range of patients.
Among surveyed hematologists, mitapivat (Agios) currently holds a slight edge in name recognition over etavopivat (Novo Nordisk). Interestingly, despite this difference in familiarity, physicians identified slightly more patients as potential candidates for etavopivat, hinting at a broader perceived clinical fit once awareness and understanding of the agent catch up. Chart audit data allows users to probe further into specific patient types which physicians perceive as better fits for either agent, as well as those who they do not perceive as candidates.
This growing interest in emerging oral therapies reflects a broader desire among hematologists for more accessible, lower-burden treatment options. While most patients are theoretically eligible for curative approaches like gene therapy, the market still favors lower-barrier alternatives. These alternatives include oral therapies that do not require referral to specialized centers or extensive pre-conditioning, and brands with which they are more familiar given other indications such as Reblozyl (Bristol Myers Squibb). Disease-modifying therapies that are easier to prescribe and integrate into routine practice have the promise to significantly improve the lives of patients who would not qualify or want gene therapy.
Access barriers compound the issue, with over half of physicians voicing frustration over payer restrictions that make it difficult to initiate optimal care. These challenges highlight a critical need – not just for clinical innovation, but also for strong manufacturer support in navigating reimbursement and expanding access.
As the beta thalassemia landscape continues to evolve, hematologists are increasingly looking toward emerging therapies that are easier to prescribe, carry fewer logistical burdens than gene therapy, and offer clear reductions in transfusion dependency. Products like mitapivat and etavopivat are well-positioned to address this gap, assuming familiarity, confidence, and access grow in tandem – but the goal to cure patients of hemoglobinopathies still carries heavy weight, with desire to treat with gene therapy consistently growing.
Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the “why” behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists’ attitudinal & demographic data to highlight differences between stated and actual treatment patterns.
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Spherix Global Insights Contacts
Sarah Hendry, Hematology Franchise Head
Sarah.Hendry@SpherixGlobalInsights.com
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