FDA Approves Winrevair, Merck’s Novel Treatment for Pulmonary Arterial Hypertension

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Merck announced that the FDA has approved Winrevair (sotatercept-csrk) to treat adults with pulmonary arterial hypertension (PAH). According to the company, this marks the first time an activin signaling inhibitor therapy, which aims to improve vascular cell proliferation balance, has been cleared for the treatment of PAH. The approval was based on promising results stemming from the Phase III STELLAR trial, showing that Winrevair increased six-minute walk distance from baseline by 41 meters at week 24, also reducing the potential for death by 84% and potential worsening of the condition.¹

“Pulmonary arterial hypertension is a rare, progressive and ultimately life-threatening disease in which blood vessels in the lungs thicken and narrow, causing significant strain on the heart,” said STELLAR trial investigator Marc Humbert, MD, PhD, professor of medicine, director, Pulmonary Hypertension Reference Center, Université Paris-Saclay, in a press release. “Based on the Phase III STELLAR trial, adding Winrevair to background PAH therapy demonstrated significant clinical benefits compared to background PAH therapy alone. This approval is an important milestone, as it offers healthcare providers a novel therapeutic option that targets a new PAH treatment pathway.”

Last week, the FDA also approved Johnson & Johnson’s Opsynvi, a combination of macitentan and tadalafil, for the treatment of PAH. This medication focuses on patients who are treatment-naïve, already on an inhibitor, or currently treated concomitantly with stable doses of macitentan 10 mg and tadalafil 40 mg as separate tablets. For this treatment, the FDA based its approval on data from the Phase III A DUE study, which found that it showed greater reduction in Pulmonary Vascular Resistance after 16 weeks versus tadalafil or macitentan monotherapy.^2,3

"Clinical guidelines recommend treating patients with initial and sequential dual-combination therapy, regardless of risk at initial diagnosis and follow-up. Historically, this required patients to take multiple pills because no single-tablet combination therapy targeting two or more pathways was available," said Kelly Chin, MD, professor of internal medicine, director, pulmonary hypertension program, UT Southwestern Medical Center, investigator in the A DUE study, in a press release. "As administration of macitentan and tadalafil together are commonly prescribed for initial therapy for PAH, the introduction of a single tablet combining both is promising for clinicians treating patients as it may help bridge the gap between clinical guidelines and everyday clinical practice, while offering a patient-friendly approach to support initial combination therapy and rapid escalation for the appropriate patients."

Other results from the STELLAR trial include:

• Improvements in FC baseline at week 24 in 29% of patients.
• An overall improvement in pulmonary vascular resistance.
• Improvement in baseline N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels.¹

“PAH remains a debilitating disease with high morbidity and mortality,” said Eliav Barr, MD, SVP, head, global clinical development, chief medical officer, Merck Research Laboratories, in a press release. “This approval of Winrevair is an important milestone and a testament to our science-led strategy and focus on the development of innovations that can help people affected by rare diseases like PAH. We are proud to bring this novel medicine to patients.”

Merck expects that Winrevair should be available in select specialty pharmacies by the end of next month.¹

References

1. FDA Approves Merck’s WINREVAIR™ (sotatercept-csrk), a First-in-Class Treatment for Adults with Pulmonary Arterial Hypertension (PAH, WHO* Group 1). Merck. March 26, 2024. Accessed March 27, 2024. https://www.merck.com/news/fda-approves-mercks-winrevair-sotatercept-csrk-a-first-in-class-treatment-for-adults-with-pulmonary-arterial-hypertension-pah-who-group-1/

2. U.S. FDA Approves OPSYNVI® (macitentan and tadalafil) as the First and Only Once-Daily Single-Tablet Combination Therapy for Patients with Pulmonary Arterial Hypertension (PAH). PR Newswire. March 22, 2024. Accessed March 27, 2024. https://www.prnewswire.com/news-releases/us-fda-approves-opsynvi-macitentan-and-tadalafil-as-the-first-and-only-once-daily-single-tablet-combination-therapy-for-patients-with-pulmonary-arterial-hypertension-pah-302097530.html

3. FDA Approves Single-Tablet Combination of Macitentan and Tadalafil for Pulmonary Arterial Hypertension. Pharmaceutical Executive. March 25, 2024. Accessed March 27, 2024. https://www.pharmexec.com/view/fda-approves-single-tablet-combination-of-macitentan-and-tadalafil-for-pulmonary-arterial-hypertension