FDA updates PAH label with trial data

In adults diagnosed with pulmonary arterial hypertension within the previous year, adding sotatercept to background therapy reduced the risk of clinical worsening events by 76% compared with placebo.

By
Kathryn Wighton
September 22, 2026
3 min

MDSpire News

Overview

The FDA has updated the sotatercept (Winrevair) label to include efficacy and safety data from the phase 3 HYPERION trial, demonstrating a 76% reduction in clinical worsening events in adults with pulmonary arterial hypertension. The trial involved 320 adults.

Background

Pulmonary arterial hypertension (PAH) is a progressive disease that can lead to significant morbidity and mortality. Understanding the efficacy and safety of new therapies, such as sotatercept, is crucial for optimizing management strategies in PAH.

Data Highlights

Outcome	Sotatercept (n=160)	Placebo (n=160)
First clinical worsening event	11% (17)	37% (59)
Risk reduction of clinical worsening events	76%	N/A
Common adverse reactions (epistaxis)	32%	7%
Common adverse reactions (telangiectasia)	26%	11%
Adverse events leading to discontinuation	3%	0%

Key Findings

The HYPERION trial included 320 adults with WHO functional class II or III PAH.
Sotatercept reduced the risk of clinical worsening events by 76% compared to placebo.
11% of patients receiving sotatercept experienced a first clinical worsening event versus 37% in the placebo group.
Common adverse reactions included epistaxis (32%) and telangiectasia (26%).
Adverse events led to treatment discontinuation in 3% of patients receiving sotatercept.

Clinical Implications

Healthcare providers should consider the updated efficacy and safety profile of sotatercept when managing patients with PAH. Monitoring for adverse reactions, particularly hemoglobin and platelet levels, is recommended during treatment initiation.