New Biologic Treatment Significantly Improves Outcomes in Severe Pulmonary Hypertension

A recent clinical trial has demonstrated that the biologic medication sotatercept, when combined with standard therapies, markedly reduces symptoms and hospitalization rates in patients with severe pulmonary arterial hypertension (PAH). Published in the New England Journal of Medicine, the study highlights that early intervention within the first year of diagnosis can lead to substantial benefits. Patients receiving sotatercept experienced a 76% decrease in the risk of disease progression, including diminished exercise capacity, worsening symptoms, and urgent hospitalizations, compared to those on placebo.

This phase 3 trial, known as HYPERION, enrolled patients diagnosed within one year of PAH onset, making it one of the first studies to focus on early-stage disease. The trial was halted early due to overwhelmingly positive outcomes, raising the ethical obligation to stop in light of clear benefits. Notably, less than 2% of patients on sotatercept required hospitalization for worsening symptoms, contrasting with 8.8% in the placebo group. The medication was generally well-tolerated, with common adverse effects being nosebleeds and spider veins.

Sotatercept works by targeting activins, proteins that are involved in the pathological remodeling of pulmonary arteries, a hallmark of PAH. By inhibiting activin signaling, the drug helps prevent artery thickening and reduces the strain on the heart.

The success of this trial builds upon earlier research, such as the STELLAR and ZENITH studies, which showed improvements in exercise capacity and survival in patients with longstanding PAH. The current findings suggest that initiating treatment early can better preserve patients’ functional status and improve long-term outcomes.

Sotatercept is approved by the U.S. Food and Drug Administration for treating PAH in adults and represents a novel approach in managing this progressive condition that often remains diagnosed at advanced stages. Early treatment could change the trajectory of the disease, providing hope for better prognosis and quality of life.

For more details, see the full study in the New England Journal of Medicine: [DOI: 10.1056/NEJMoa2508170] (source: https://medicalxpress.com/news/2025-09-biologic-drug-symptoms-hospitalization-severe.html).