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News
Sotatercept (Winrevair) is being used off-label in pulmonary hypertension patients beyond its FDA-approved Group 1 PAH indication — and new real-world data suggest its safety profile largely holds up. A study of 590 patients found no significant difference in mortality or serious bleeding risk between Group 2/3 PH and Group 1 PAH recipients, though hospitalization and ER visit rates were higher in the off-label group.
Sotatercept (Winrevair) was FDA-approved in March 2024 specifically for Group 1 pulmonary arterial hypertension (PAH), but real-world clinicians are already using it well beyond that boundary. A new study using the TriNetX Collaborative Research Network found that 70% of the 590 patients who received a sotatercept dispense had Group 2/3 PH — conditions involving left heart disease and/or lung disease — rather than the approved Group 1 PAH indication. The findings were presented at the European Respiratory Society International Congress.
The reassuring part: adjusted risks for mortality, GI/serious hemorrhage, and thrombocytopenia did not significantly differ between the off-label Group 2/3 PH group and the approved Group 1 PAH group. However, patients with Group 2/3 PH did face significantly higher risks for emergency department visits and all-cause hospitalization, likely reflecting their greater baseline disease burden rather than drug toxicity.
Key Takeaways:
Why it matters: These findings offer reassurance that Winrevair's safety profile is broadly consistent across PH phenotypes in routine practice — even in sicker, more complex patients. As the PH field moves toward a biology-based rather than classification-based treatment approach, this real-world evidence could help shape future guidelines and trial design.
