Etavopivat

The FDA pushed back its decision date by three months for Agios Pharmaceuticals’ drug Pyrukynd in thalassemia, the Cambridge, MA-based company announced Thursday morning. The agency’s decision was expected by Sept. 7, but that deadline has now been moved to Dec. 7. Agios said it submitted a proposal for a drug safety monitoring program called REMS to reduce the risk of liver injury with Pyrukynd, resulting in the three-month delay. Last year, Agios reported that two of 301 patients who received the drug in a trial for transfusion-dependent thalassemia had liver damage in the first six months on the therapy, which led them to stop treatment. Agios’ shares $AGIO fell 17% Thursday premarket. Thalassemia is a genetic blood disorder that interferes with a person’s ability to make healthy red blood cells. Some patients require regular blood transfusions to live, while others don’t — though those patients may need transfusions at times. The biotech is seeking approval of Pyrukynd in all adult patients with thalassemia after studying the drug in transfusion-dependent and transfusion-independent patients with either alpha or beta thalassemia. An approval would mark a key expansion for the drug, which is also known as mitapivat. It was previously approved to treat a rare condition called pyruvate kinase deficiency. In August, Pyrukynd was cleared by Saudi Arabia for thalassemia, its first approval in the disease. The company is also expecting results from a Phase 3 sickle cell study later this year. In the second quarter, Agios reported that Pyrukynd generated $12.5 million in sales. Wall Street analysts believe that the drug can become a multibillion-dollar product after adding thalassemia and sickle cell indications. Agios’ key competitor is Novo Nordisk, which acquired a similar drug called etavopivat through its $1.1 billion purchase of Forma Therapeutics in 2022. Novo is also studying etavopivat in late-stage trials with sickle cell disease and thalassemia patients.

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 (GLOBE NEWSWIRE) -- 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. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix’s unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit spherixglobalinsights.com or connect through LinkedIn . For more details on Spherix’s primary market research reports and interactive dashboard offerings, visit or register here: https://clientportal.spherixglobalinsights.com NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight’s analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 sarah.hendry@spherixglobalinsights.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.