Sotatercept Gets Priority Review for Pulmonary Aterial Hypertension

The Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for sotatercept for the treatment of adults with pulmonary arterial hypertension (PAH) (WHO Group 1). 

Sotatercept is a first-in-class activin receptor type IIA-Fc fusion protein. The application is supported by 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 and clinically meaningful improvement in 6MWD among patients treated with sotatercept compared with placebo (34.4m vs 1.0m, respectively; Hodges-Lehmann estimated difference, 40.8m [95% CI, 27.5-54.1]; P <.001). 

Sotatercept was also associated with statistically significant improvements in 8 out of 9 secondary outcome measures, including the proportion of participants achieving multicomponent improvement (defined as improvement in 6MWD, improvement in N-terminal pro-B-type natriuretic peptide level, and either improvement in WHO functional class [FC] or maintenance of WHO FC II), and the outcome measure of time to death or the first occurrence of a clinical worsening event. An assessment of the Cognitive/Emotional Impacts domain score of PAH-SYMPACT^® did not result in a statistical significant change.

Compared with placebo, the following adverse events occurred more frequently with sotatercept: epistaxis, dizziness, telangiectasia, increased hemoglobin levels, thrombocytopenia, and increased blood pressure.

“Despite advances in the treatment of PAH over the last 2 decades, there is still a significant need to improve outcomes for patients,” said Dr Joerg Koglin, senior vice president, global clinical development, Merck Research Laboratories. “The FDA’s acceptance of this application is an exciting milestone in our journey to bring this novel activin signaling inhibitor to patients. Based on the profound improvements across primary and secondary outcome measures in the phase 3 STELLAR trial, we believe sotatercept has the potential to transform the treatment of patients with PAH.”

A Prescription Drug User Fee Act target date of March 26, 2024 has been set for the application.

This article originally appeared on MPR