Marius Hoeper, MD, Respiratory Physician at Hannover Medical School, Germany, discusses a recent regulatory update by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in the treatment of pulmonary arterial hypertension (PAH).
PAH is characterized by abnormally high blood pressure in the pulmonary artery. Symptoms include shortness of breath during exercise and fainting spells. The symptoms tend to get worse over time and may include dizziness, swelling of the ankles or legs, chest pain, and a racing pulse. Some cases of PAH are due to genetic changes in the BMPR2 gene. PAH can also occur secondary to underlying conditions such as connective tissue diseases, HIV infection, chronic hemolytic anemia, and congenital heart disease. PAH can also be induced by certain drugs and toxins.
The European Medicines Agency’s CHMP recommended the approval of an expanded indication for Winrevair (sotatercept), in combination with other PAH therapies, for the treatment of PAH in adults with WHO Functional Class II, III, and IV. This recommendation is based on results from the phase 3 ZENITH clinical trial (NCT04896008).
ZENITH was a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial that enrolled 172 adult participants with PAH (WHO Functional Class III or IV) at high risk of mortality. Sotatercept is an activin signaling inhibitor treatment that improves the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation approved for the treatment of PAH.
The trial demonstrated that sotatercept, in addition to background therapy, resulted in a statistically significant and clinically meaningful 76% reduction in the risk of major morbidity and mortality outcomes in adults with PAH WHO functional class III or IV compared to placebo. Time to first occurrence of all-cause death, lung transplantation or PAH-worsening hospitalization of 24 hours or more occurred in 15 sotatercept-treated participants versus 47 placebo-treated participants.
Sotatercept is currently approved in the European Union for adults with PAH with WHO Functional Class II to III, to improve exercise capacity. The CHMP recommendation will now be reviewed by the European Commission with a final decision expected in the first quarter of 2026.
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To learn more about PAH and other rare lung diseases, visit https://checkrare.com/diseases/lung-diseases/
