From the FDA Drug Label
HIGHLIGHTS OF PRESCRIBING INFORMATION These highlights do not include all the information needed to use WINREVAIR safely and effectively. See full prescribing information for WINREVAIR. WINREVAIR™ (sotatercept-csrk) for injection, for subcutaneous use Initial U. S. Approval: 2024 INDICATIONS AND USAGE WINREVAIR is an activin signaling inhibitor indicated for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1) to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events.
The main highlights for the trial with sotatercept are:
- Indication: Sotatercept is indicated for the treatment of adults with pulmonary arterial hypertension (PAH, WHO Group 1) to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events.
- Dosage: The recommended starting dose is 0.3 mg/kg by subcutaneous injection, with a recommended target dose of 0.7 mg/kg every 3 weeks.
- Key warnings and precautions include erythrocytosis, severe thrombocytopenia, serious bleeding, embryo-fetal toxicity, and impaired fertility.
- Common adverse reactions include headache, epistaxis, rash, telangiectasia, diarrhea, dizziness, and erythema. 1 1
From the Research
Sotatercept is a promising investigational therapy for the treatment of anemia in patients with lower-risk myelodysplastic syndromes, as evidenced by a phase 2, dose-ranging trial published in The Lancet. Haematology in 2018 2.
Key Highlights
- The trial demonstrated that sotatercept was well tolerated and effective in improving hemoglobin levels and reducing transfusion burden in patients with lower-risk myelodysplastic syndromes.
- The primary efficacy endpoint was the proportion of patients who achieved hematological improvement-erythroid (HI-E), according to International Working Group 2006 criteria.
- The most commonly reported adverse events were fatigue and peripheral edema.
Study Details
- The study was an open-label, multicentre, dose-ranging, phase 2 trial that took place at 11 treatment centres in the USA and France.
- Patients were randomly assigned to receive either 0.1 or 0.3 mg/kg sotatercept subcutaneously, using a permuted-block method with stratification for serum erythropoietin concentration and transfusion burden.
- The trial showed that 36 (49%) of 74 patients achieved HI-E, with 29 (47%) of 62 patients with a high transfusion burden achieving HI-E.
Additional Evidence
- A phase II, open-label, dose-finding study published in Haematologica in 2019 found that sotatercept was effective and well tolerated in patients with β-thalassemia, with 18 (60%) of 30 non-transfusion-dependent β-thalassemia patients achieving a mean hemoglobin increase of ≥1.0 g/dL sustained for ≥12 weeks 3.
- Another study published in Kidney international reports in 2019 found that sotatercept exhibited a favorable safety profile and was associated with trends in dose-related slowing of vascular calcification in patients with end-stage kidney disease (ESKD) 4.
Recommendation
Based on the available evidence, sotatercept appears to be a promising treatment option for anemia in patients with lower-risk myelodysplastic syndromes and other conditions, and its use should be considered in clinical practice, particularly in patients who have failed or are ineligible for erythropoiesis-stimulating agents (ESAs) 2, 3, 4.