The US Food and Drug Administration (FDA) has approved an update to the US label for WINREVAIR (sotatercept-csrk), Merck’s activin signaling inhibitor for pulmonary arterial hypertension, to include efficacy and safety data from the Phase 3 HYPERION trial. The trial specifically evaluated adults newly diagnosed with pulmonary arterial hypertension — within 12 months of screening, World Health Organization functional class II or III, at intermediate-to-high risk of disease progression — adding WINREVAIR on top of standard background therapy.
Adding WINREVAIR reduced the risk of clinical worsening events by 76 percent compared to placebo (only 10.6 percent of WINREVAIR patients experienced a first worsening event, versus 36.9 percent on placebo).
On safety, the most common side effects were nosebleeds, small vascular skin lesions called telangiectasia, and increased hemoglobin — broadly consistent with earlier trials. The label update also reinforces existing precautions: regular monitoring of hemoglobin and platelets is required, since the drug can raise red blood cell counts, which risks blood clots, or lower platelets, which risks bleeding.
This news arrives alongside newly published European Respiratory Society clinical guidelines for treatment of pulmonary arterial hypertension — the first formal guidelines to include WINREVAIR. The drug received a “strong” recommendation for patients on background therapy who haven’t reached low-risk status, with the underlying evidence rated “high” certainty, the top of a four-level scale. See also our article at this link
Read the company press release here