Winrevair Approved for Pulmonary Arterial Hypertension – Renal and Urology News

The Food and Drug Administration (FDA) has approved Winrevair^™ (sotatercept-csrk) 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. 

Winrevair is a first-in-class recombinant activin receptor type IIA-Fc fusion protein. It is designed to bind to activin A and other TGF-β superfamily ligands resulting in improved balance between the pro-proliferative and anti-proliferative signaling to modulate vascular proliferation.

The approval was based on data from the double-blind, placebo-controlled phase 3 STELLAR study (ClinicalTrials.gov Identifier: NCT04576988), which evaluated the efficacy and safety of sotatercept in 323 adult patients with PAH (WHO Group 1). Study participants who were receiving stable background PAH therapy were randomly assigned 1:1 to receive either sotatercept or placebo administered subcutaneously every 3 weeks. 

The primary endpoint was exercise capacity, which was measured by the change from baseline in 6-minute walk distance (6MWD) at 24 weeks. Results showed a statistically significant improvement in 6MWD among patients treated with sotatercept vs placebo with a Hodges-Lehmann estimated difference of 41m (95% CI, 28-54; P <.001).

Additionally, 29% of patients treated with sotatercept achieved an improvement from baseline by at least 1 WHO FC at 24 weeks vs placebo (P <.001). Treatment with sotatercept also achieved an 84% reduction in the occurrence of death from any cause or PAH clinical worsening events vs placebo (5.5% vs 26.3%, respectively; hazard ratio [HR], 0.16 [95% CI, 0.08-0.35]; P <.001).

The prescribing information for Winrevair carries warnings and precautions associated with erythrocytosis, severe thrombocytopenia, serious bleeding, and embryo-fetal toxicity. The most common adverse reactions (incidence ≥10% and 5% more than placebo) for Winrevair are headache, epistaxis, rash, telangiectasia, diarrhea, dizziness, and erythema.

Winrevair is supplied as a 45mg or 60mg lyophilized cake or powder in a single-dose vial for subcutaneous injection after reconstitution, and is expected to be available in select specialty pharmacies by the end of April.

“New treatment options continue to be needed for patients with pulmonary arterial hypertension that support important clinical goals, including increasing exercise capacity and improving functional class,” said Dr Aaron Waxman, Executive Director of the Center for Pulmonary Heart Diseases at Brigham and Women’s Hospital and investigator on the Phase 3 STELLAR study. “Sotatercept added to background therapy has the potential to become a new standard of care option for patients with pulmonary arterial hypertension.”

The FDA previously granted Priority Review, Breakthrough Therapy, and Orphan Drug designations to sotatercept for this indication.

This article originally appeared on MPR