Positive CHMP opinion for Winrevair (sotatercept) in pulmonary arterial hypertension (PAH).- Merck Inc.,

Winrevair was previously granted Priority Medicines (PRIME) and orphan designation by the EMA for the treatment of PAH. The European Commission (EC) will now review the CHMP recommendation, and the EC’s decision on the marketing authorization application of Winrevair in the EU, Iceland, Liechtenstein and Norway is expected in the third quarter of 2024.

"PAH is a progressive and debilitating rare disease,” said Dr. Joerg Koglin, senior vice president and head of general medicine, global clinical development, Merck Research Laboratories. “There is still a significant need for new therapies for patients. This positive opinion marks the first step toward expanding access to our first-in-class activin signaling inhibitor therapy, Winrevair, for eligible adults with PAH in Europe. We are pleased with the CHMP recommendation and look forward to the European Commission’s decision.”

Winrevair is administered once every 3 weeks as a single injection under the skin and may be administered by patients or caregivers with guidance, training and follow-up from a healthcare provider.

The CHMP recommendation is based on data from the Phase III STELLAR trial of Winrevair on top of background PAH therapy compared to background therapy alone. Winrevair demonstrated a statistically significant and clinically meaningful improvement in 6-minute walk distance, the study’s primary endpoint, and on multiple important secondary outcome measures, including reducing the risk of death from any cause or PAH clinical worsening events. These results were published in The New England Journal of Medicine.

“Winrevair is the first activin signaling inhibitor therapy and is proposed to modulate the vascular proliferation underlying PAH,” said Dr. Marius Hoeper, Hannover Medical School, Germany. “Based on the clinical benefits demonstrated in primary and secondary outcome measures in the Phase III STELLAR study, Winrevair has the potential to play a critical role in the treatment of appropriate adults with PAH. It is very encouraging that physicians in Europe may soon have a novel treatment option available that targets a new PAH treatment pathway.

See- ORIGINAL ARTICLE. VOL. 388 NO. 16APR 20, 2023CMEE "Phase III Trial of Sotatercept for Treatment of Pulmonary Arterial Hypertension",. M.M. Hoeper and Others. N Engl J Med 2023;388:1478-1490.