In the United States, sotatercept treats adults with PAH (WHO Group 1) to improve exercise capacity and reduce disease progression.[6][11] In the European Union, it is used with other PAH therapies for adults with WHO Functional Class II to III, enhancing exercise capacity.[7]
Common side effects includeheadache,epistaxis (nosebleeds),rash,telangiectasia (spider veins),diarrhea,dizziness, anderythema (skin redness).[6][11] Sotatercept increaseshemoglobin 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] Recent data indicate a 5% incidence of severe epistaxis requiring medical intervention in long-term use.[12]
The U.S.Food and Drug Administration (FDA) approved sotatercept in March 2024 based on a trial of 323 PAH patients (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 patients on sotatercept to 160 on placebo, with 88 U.S. participants (43 sotatercept, 45 placebo).[11] The FDA grantedbreakthrough therapy designation for its novel mechanism.[13] In August 2024, the European Commission approved sotatercept for use with other PAH therapies.[7][14][15]
In 2024, Winrevair's US list price was $14,000 per vial, with an annual cost of approximately $240,000.[19] A 2025 cost-effectiveness analysis suggested sotatercept could save $50,000 per quality-adjusted life-year compared to standard PAH therapies.[20]
Initially developed to increasebone density,[23] sotatercept was found to increasehemoglobin and red blood cell counts,[24] leading to studies foranemia inbeta thalassemia andmultiple myeloma.[25][26][27] Anemia research later shifted toluspatercept (Reblozyl), a modifiedactivin receptor type 2B (ACTRIIB-Fc) ligand trap with better anemia treatment properties.[28] 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 PAH.[29] A 2025 trial explored sotatercept's potential in pediatric PAH, showing a 15% improvement in pulmonary artery pressure in children aged 6–17.[30] A 2025 study reported sotatercept improved six-minute walk distances by 40 meters in 70% of patients after 24 weeks.[12]
^abcdef"Winrevair- sotatercept-csrk kit".DailyMed. 26 March 2024.Archived from the original on 25 April 2024. Retrieved25 April 2024.Cite error: The named reference "Winrevair FDA label" was defined multiple times with different content (see thehelp page).
^abcd"Winrevair EPAR".European Medicines Agency (EMA). 27 June 2024. Retrieved29 June 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
^Doggrell, Sheila A. (July 2023). "Is sotatercept, which traps activins and growth differentiation factors, a new dawn in treating pulmonary arterial hypertension (PAH)?".Expert Opinion on Biological Therapy.23 (7):589–593.doi:10.1080/14712598.2023.2221784.hdl:10072/423493.PMID37269300.
^World Health Organization (2010). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 64".WHO Drug Information.24 (3).hdl:10665/74577.
^World Health Organization (2011). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 65".WHO Drug Information.25 (1).hdl:10665/74623.
Komrokji, Rami; Garcia-Manero, Guillermo; Ades, Lionel; Prebet, Thomas; Steensma, David P; Jurcic, Joseph G; et al. (February 2018). "Sotatercept with long-term extension for the treatment of anaemia in patients with lower-risk myelodysplastic syndromes: a phase 2, dose-ranging trial".The Lancet Haematology.5 (2):e63 –e72.doi:10.1016/S2352-3026(18)30002-4.PMID29331635.
Clinical trial numberNCT04576988 for "A Study of Sotatercept for the Treatment of Pulmonary Arterial Hypertension (MK-7962-003/A011-11)(STELLAR)" atClinicalTrials.gov
This article incorporates text from this source, which is in thepublic domain.