FDA approves Sotatercept, first-in-class treatment for adults with PAH

Sotatercept (Winrevair; Merck) has been approved by the FDA for the treatment of patients with pulmonary arterial hypertension (PAH), according to a press release from Merck.1 The approval comes after sotatercept was granted priority review in September 2023.2 The treatment also received FDA breakthrough therapy and orphan drug designations for the 45 mg and 60 mg doses.

PAH is a life-threatening disease that causes blood vessels in the lungs to narrow and increase strain on the heart, and can lead to heart failure, reduced physical activity, and reduced life expectancy , among other adverse effects. The 5-year mortality rate for patients with PAH is about 43%, with approximately 40,000 people in the United States living with the disease.

Subtercept | Image credit: Merck

Sotatercept is an activin signaling inhibitor that has been found to increase exercise capacity, improve WHO functional class, and reduce the risk of clinical worsening in PAH patients when added to therapy standard base.1 The approval of sotatercept is the first for an activin signaling inhibitor for PAH.

The approval is based on data from the multicenter, double-blind, phase 3 STELLAR trial, with results published in The New England Journal of Medicine in April 2023.3 The trial allocated all participants 1:1 to receive either sotatercept (n = 163) or placebo (n = 160) every 3 weeks in addition to standard of care PAH therapy. Change in 6-minute walk distance (6MWD) from baseline to week 24 was the primary endpoint of the study.

The researchers found a mean change of 34.4 m (95% CI, 33.0-35.5) from baseline for 6MWD at week 24 in the subtercept group versus a change mean 1.0 m (95% CI, 0.3 to 3.5) in 6MWD for placebo. group The estimated difference in 6MWD after 24 weeks between the sotatercept and placebo groups was 40.8 m (95% CI, 27.5–54.1).

New treatment options for patients with pulmonary arterial hypertension that support important clinical goals, such as increasing exercise capacity and improving functional class, continue to be needed. Sotatercept added to background therapy has the potential to become a new standard of care option for patients with pulmonary arterial hypertension. , he said in a statement.1

Secondary endpoints, tested hierarchically, were multicomponent improvement, change in pulmonary vascular resistance, change in N-terminal pro-B-type natriuretic peptide level, improvement in World Organization functional class of Health, time to death or clinical worsening, French risk score. , and changes in scores for the Pulmonary Arterial Hypertension-Symptoms and Impact (PAH-SYMPACT) Physical Impacts, Cardiopulmonary Symptoms and Cognitive/Emotional Impacts domain scores. Except for the PAH-SYMPACT Cognitive/Emotional Impacts domain score, all secondary endpoints were significantly improved with sotatercept compared with placebo; were assessed at week 24 except for time to death or clinical worsening.

Adverse effects of sotatercept include epistaxis, telangiectasia, dizziness, headache, rash, thrombocytopenia, increased hemoglobin, and increased blood pressure. These occurred more often in the sotatercept group compared to placebo. Healthcare professionals should also monitor hemoglobin and platelets prior to a dose of sotatercept for the first 5 doses given these potential adverse effects.

We are excited to see industry research lead to a better understanding of PAH and the development of a drug in a new treatment pathway that expands options for the patient community,” said Matt Granato, president and CEO of the Pulmonary Hypertension Association.1

The medication will be available to all patients starting in April, the company said in the press release.


  1. FDA approves Merck’s WINREVAIR (sotatercept-csrk), a first-in-class treatment for adults with pulmonary arterial hypertension (PAH, WHO group 1). News release. Merck; March 26, 2024. Retrieved March 26, 2024.
  2. Merck receives FDA priority review for new biologics license application for sotatercept, an activin signaling inhibitor to treat adults with pulmonary arterial hypertension. News release. Merck. September 28, 2023. Accessed March 22, 2024. https://www.merck.com/news/merck-receives-priority-review-from-fda-for-new-biologics-license-application-for- sotatercept-an -activin-signaling-inhibitor-to-treat-adults-with-pulmonary-arterial-hypertension-pah/
  3. Hoeper MM, Badesch DB, Ghofrani A, et al. Phase 3 trial of sotatercept for the treatment of pulmonary arterial hypertension. N Engl J Med. 2023;388(16):1478-1490. doi:10.1056/NEJMoa2213558

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