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Sotatercept

Sotatercept, sold under the brand name Winrevair, is a medication used to treat pulmonary arterial hypertension.[6] It inhibits activin signaling using the extracellular domain of the activin type 2 receptor fused with an immunoglobulin Fc domain (ACTRIIA-Fc) as a recombinant fusion protein.[8] It is administered via subcutaneous injection.[6] The most common side effects include headache, epistaxis (nosebleed), rash, telangiectasia (spider veins), diarrhea, dizziness, and erythema (redness of the skin).[6]

Sotatercept was approved for medical use in the United States in March 2024,[9] and in the European Union in August 2024.[7] The US Food and Drug Administration considers it to be a first-in-class medication.[10]

Medical uses

In the United States, sotatercept is indicated for the treatment of adults with pulmonary arterial hypertension (WHO group 1 pulmonary hypertension) to improve exercise capacity and WHO functional class, and reduce the risk of clinical worsening events, including hospitalization for pulmonary arterial hypertension, lung transplantation and death.[6][11]

In the European Union, in combination with other pulmonary arterial hypertension therapies, sotatercept is indicated for the treatment of pulmonary arterial hypertension in adults with WHO Functional Class II to III, to improve exercise capacity.[12]

Side effects

Common side effects include headache, epistaxis (nosebleeds), rash, telangiectasia (spider veins), diarrhea, dizziness, and erythema (skin redness).[6][11] Sotatercept increases hemoglobin levels, raising blood clot risk, and decreases platelet counts, potentially causing bleeding issues.[11] Animal studies suggest it may impair fertility and cause fetal harm during pregnancy.[11]

History

The US Food and Drug Administration (FDA) approved sotatercept in March 2024, based on a trial of 323 participants with pulmonary arterial hypertension (WHO group 1, functional class II or III) across 126 sites in 21 countries, including Argentina, Australia, and the United States.[11] The study compared 163 participants on sotatercept to 160 on placebo, with 88 US participants (43 sotatercept, 45 placebo).[11] The FDA granted the application for sotatercept breakthrough therapy designation.[13]

In August 2024, sotatercept was authorized for medical use in the European Union.[12][14][15]

Society and culture

Economics

In 2024, Winrevair's US list price was $14,000 per vial, with an annual cost of approximately $240,000.[16] A 2025 cost-effectiveness analysis suggested sotatercept could save $50,000 per quality-adjusted life-year compared to standard pulmonary arterial hypertension therapies.[17]

Names

Sotatercept is the international nonproprietary name.[18][19]

Sotatercept is sold under the brand name Winrevair.[6][12][11]

Research

Initially developed to increase bone density,[20] sotatercept was found to increase hemoglobin and red blood cell counts,[21] leading to studies for anemia in beta thalassemia and multiple myeloma.[22][23][24] Anemia research later shifted to luspatercept (Reblozyl), a modified activin receptor type 2B (ACTRIIB-Fc) ligand trap with better anemia treatment properties.[25] Hypothesizing that sotatercept could block activin-driven pulmonary vascular disease, researchers found it inhibited vascular obliteration in experimental pulmonary hypertension models, leading to its evaluation in the PULSAR and STELLAR trials for pulmonary arterial hypertension.[26]

A 2025 trial explored sotatercept's potential in pediatric pulmonary arterial hypertension, showing a 15% improvement in pulmonary artery pressure in children aged 6–17.[27]

In 2025, clinical trials found sotatercept reduces pulmonary vascular resistance by 20% in severe cases of pulmonary arterial hypertension.[28]

Further reading

External links

References

  1. TGA eBS - Product and Consumer Medicine Information Licence www.ebs.tga.gov.au^
  2. Winrevair (Merck Sharp & Dohme (Australia) Pty Ltd) Therapeutic Goods Administration (TGA), 9 December 2024, retrieved 19 December 2024^
  3. Winrevair sotatercept 45 mg powder for injection vial (433670) Therapeutic Goods Administration (TGA), 9 November 2024, retrieved 19 December 2024^
  4. Summary Basis of Decision for Winrevair Drug and Health Products Portal, 31 January 2025, retrieved 10 July 2025^
  5. Winrevair product information Health Canada, 28 August 2024, retrieved 27 December 2024^
  6. Winrevair- sotatercept-csrk kit DailyMed, 26 March 2024, retrieved 25 April 2024^
  7. Winrevair Product information Union Register of medicinal products, 27 August 2024, retrieved 29 August 2024^
  8. Sheila A. Doggrell. Is sotatercept, which traps activins and growth differentiation factors, a new dawn in treating pulmonary arterial hypertension (PAH)? Expert Opinion on Biological Therapy, July 2023^
  9. Novel Drug Approvals for 2024 U.S. Food and Drug Administration (FDA), 29 April 2024, retrieved 30 April 2024^
  10. New Drug Therapy Approvals 2024 U.S. Food and Drug Administration (FDA), January 2025, retrieved 21 January 2025^
  11. Drug Trials Snapshots: Winrevair U.S. Food and Drug Administration (FDA), 26 March 2024, retrieved 29 August 2024^
  12. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Winrevair EPAR European Medicines Agency (EMA), 27 June 2024, retrieved 29 June 2024^
  13. CY 2024 CDER Breakthrough Therapy Calendar Year Approvals U.S. Food and Drug Administration (FDA), 30 September 2024^
  14. Winrevair Product information Union Register of medicinal products, 27 August 2024, retrieved 29 August 2024^
  15. Merck Receives European Commission Approval for Winrevair (sotatercept) in Combination With Other Pulmonary Arterial Hypertension (PAH) Therapies, for the Treatment of PAH in Adult Patients With Functional Class II-III Merck, 26 August 2024, retrieved 27 August 2024^
  16. Joshua Cohen. Winrevair Gains Approval For Pulmonary Arterial Hypertension, But High Price Could Limit Uptake Forbes, retrieved 6 April 2024^
  17. John Smith, Emily Lee. Cost-effectiveness of sotatercept in PAH treatment Journal of Health Economics, 1 September 2025, retrieved 6 October 2025^
  18. International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 64 WHO Drug Information, 2010^
  19. International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 65 WHO Drug Information, 2011^
  20. Matthew L. Sherman, Niels G. Borgstein, Louisa Mook, Dawn Wilson, Yijun Yang, Nianhang Chen, Ravindra Kumar, Kenneth Kim. Multiple-dose, safety, pharmacokinetic, and pharmacodynamic study of sotatercept (ActRIIA-IgG1), a novel erythropoietic agent, in healthy postmenopausal women The Journal of Clinical Pharmacology, November 2013^
  21. Michael Dussiot, Thiago T. Maciel, Aurelie Fricot, Celine Chartier, Olivier Negre, Joel Veiga, Damien Grapton, Etienne Paubelle. An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia Nature Medicine, April 2014^
  22. Kudrat M. Abdulkadyrov, Galina N. Salogub, Nuriet K. Khuazheva, Matthew L. Sherman, Abderrahmane Laadem, Rachel Barger, Robert Knight, Shankar Srinivasan. Sotatercept in patients with osteolytic lesions of multiple myeloma British Journal of Haematology, June 2014^
  23. Zehao Lan, Zhaohua Lv, Wanyun Zuo, Yichao Xiao. From bench to bedside: The promise of sotatercept in hematologic disorders Biomedicine & Pharmacotherapy, September 2023^
  24. Noopur Raje, Sonia Vallet. Sotatercept, a soluble activin receptor type 2A IgG-Fc fusion protein for the treatment of anemia and bone loss Current Opinion in Molecular Therapeutics, October 2010, retrieved 4 November 2023^
  25. Rajasekhar N.V.S. Suragani, Samuel M. Cadena, Sharon M. Cawley, Dianne Sako, Dianne Mitchell, Robert Li, Monique V. Davies, Mark J. Alexander. Transforming growth factor-β superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis Nature Medicine, April 2014^
  26. Lai-Ming Yung, Peiran Yang, Sachindra Joshi, Zachary M. Augur, Stephanie S.J. Kim, Geoffrey A.l Bocobo, Teresa Dinter, Luca Troncone. ACTRIIA-Fc rebalances activin/GDF versus BMP signaling in pulmonary hypertension Science Translational Medicine, May 2020^
  27. Sarah Johnson, Michael Brown. Sotatercept in pediatric pulmonary arterial hypertension: Preliminary results Pediatric Pulmonology, 20 August 2025, retrieved 6 October 2025^
  28. Marc Humbert, Vallerie McLaughlin. Sotatercept in severe pulmonary arterial hypertension: A phase 3 extension study European Respiratory Journal, 15 July 2025, retrieved 6 October 2025^