FDA Approves New PAH Treatments: OPSYNVI® and Winrevair

The FDA has approved two groundbreaking treatments for Pulmonary Arterial Hypertension (PAH): OPSYNVI®, a once-daily tablet combining macitentan and tadalafil from Johnson & Johnson, and Merck's Winrevair (sotatercept-csrk), a first-in-class therapy. Both treatments offer significant advancements in PAH management, aligning with clinical recommendations for early combination therapy. These approvals highlight the FDA's commitment to offering more streamlined and effective management options for patients with this challenging condition.

The following article originally appeared in Cardiovascular Business.

The U.S. Food and Drug Administration (FDA) has approved two new medications for pulmonary arterial hypertension (PAH), a rare vascular condition that often leads to heart failure.

FDA approves new single-tablet combination of macitentan and tadalafil

First, the agency approved Johnson & Johnson’s single-tablet combination of macitentan and tadalafil, which is being sold under the brand name Opsynvi. The FDA’s decision was largely based on data from the A DUE clinical trial, which found that treatment with Opsynvi was linked to considerable improvements in pulmonary vascular resistance after 16 weeks compared to either macitentan or tadalafil on their own. Initial A DUE results were presented at ACC.23 in New Orleans.

Kelly Chin, MD, professor of internal medicine and director of the pulmonary hypertension program at UT Southwestern Medical Center, was an investigator of the A DUE study. Chin discussed the FDA approval in a new statement.

“Clinical guidelines recommend treating patients with initial and sequential dual-combination therapy, regardless of risk at initial diagnosis and follow-up,” Chin said. “Historically, this required patients to take multiple pills because no single-tablet combination therapy targeting two or more pathways was available. 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.”

“We're thrilled to bring this single-tablet combination therapy to patients, as it has the potential to optimize disease management and fulfill a significant unmet need in supporting recently updated treatment guidelines that call for initial or early combination treatment,” added James F. List, MD, PhD, global therapeutic area head of Johnson & Johnson.

FDA approves sotatercept-csrk injections

The FDA has also approved sotatercept-csrk, Merck’s new injectable treatment for PAH. Sold under the brand name Winrevair, the drug is approved to increase exercise capacity, improve World Health Organization functional class and reduce the risk of “clinical worsening events.”

Merck noted that Winrevair is the first FDA-approved drug of its class; it was designed to modify PAH in patients as opposed to improving disease management. The FDA’s approval was based on data from STELLAR, a clinical trial that evaluated the the safety and effectiveness of sotatercept-csrk in 163 patients. Overall, adding the drug to background therapy was associated with improvements in six-minute walk distance and multiple other secondary outcomes.

“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,” STELLAR investigator Marc Humbert, MD, PhD, professor of medicine and director of the Pulmonary Hypertension Reference Center at the Université Paris-Saclay, said in a prepared statement. “Based on the Phase 3 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.”

“A diagnosis of PAH is a life-changing experience for patients and families due to its chronic, progressive nature,” Pulmonary Hypertension Association President Matt Granato said in the same statement. “Patients with PAH experience limiting symptoms such as shortness of breath and fatigue. We are excited to see industry research leading to a better understanding of PAH and the development of a medicine in a novel treatment pathway that expands options for the patient community.”

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