FDA Approves Merck’s Winrevair for Adults with Pulmonary Arterial Hypertension

Rare Daily Staff

The U.S. Food and Drug Administration Merck’s Winrevair for the treatment of adults with pulmonary arterial hypertension to increase exercise capacity, improve World Health Organization functional class, and reduce the risk of clinical worsening events.

Pulmonary arterial hypertension (PAH) 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. Due to its chronic, progressive nature, patients with PAH experience limiting symptoms such as shortness of breath and fatigue.

Winrevair (sotatercept-csrk) is the first FDA-approved activin signaling inhibitor therapy for PAH, representing a new class of therapy that works by improving the balance between pro- and anti-proliferative signaling to regulate vascular cell proliferation underlying PAH.

“Based on the Phase 3 STELLAR trial, adding Winrevair to background PAH therapy demonstrated significant clinical benefits compared to background PAH therapy alone,” said Marc Humbert, professor of Medicine and director of the Pulmonary Hypertension Reference Center at the Université Paris-Saclay and investigator on the phase 3 STELLAR study. “This approval is an important milestone, as it offers healthcare providers a novel therapeutic option that targets a new PAH treatment pathway.”

The approval is based on the phase 3 STELLAR trial, a global, double-blind, placebo-controlled, multicenter, parallel-group clinical trial of 323 patients with PAH, which compared Winrevair (n=163) to placebo (n=160), both in combination with background standard of care therapies in adult patients with PAH. Results showed adding Winrevair to background therapy increased six-minute walk distance from baseline by 41 meters at Week 24 and significantly improved multiple important secondary outcome measures, including reducing the risk of death from any cause or PAH clinical worsening events by 84 percent versus background therapy alone.

Winrevair is given once every three weeks by subcutaneous injection and may be administered by appropriate patients or caregivers with guidance, training, and follow-up from a healthcare provider. Merck estimates that Winrevair will be available for dispensing by select specialty pharmacies in the U.S. by the end of April.

“PAH remains a debilitating disease with high morbidity and mortality,” said Eliav Barr, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. “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.”

Winrevair is the subject of a licensing agreement with Bristol Myers Squibb.

Photo: Eliav Barr, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories