Pulmonary arterial hypertension drug studied at UC receives FDA approval

Clinical research conducted at UC Health and the University of Cincinnati College of Medicine played a role in the U.S. Food and Drug Administration (FDA)'s March 26 approval of WINREVAIR™ (sotatercept-csrk) for the treatment of pulmonary arterial hypertension (PAH) in adults.

Merck has developed WINREVAIR™, a pioneering biologic fusion protein and the sole FDA-approved therapy for pulmonary arterial hypertension (PAH) that specifically targets the signaling pathways of activin and GDF proteins. As an innovative approach in PAH therapy, it aims to recalibrate the signaling within blood vessels, thereby regulating vascular cell growth and offering potential for the reversal of PAH's foundational vascular changes.

Merck & Co's Dedication to PAH Research

By focusing on innovative research and global clinical development, they aim to introduce therapies that address unmet needs within the PAH community, reaffirming their commitment to improving patient outcomes worldwide.

"We haven't had a new pathway drug for PAH that's shown promise in nearly a decade," said Arun Jose, MD, Assistant Professor of Internal Medicine in the UC College of Medicine's Division of Pulmonary, Critical Care and Sleep Medicine and a UC Health physician.

What is Pulmonary Arterial Hypertension?

Pulmonary arterial hypertension (PAH), a chronic and progressive vascular disease affecting the lungs, leads to a life-threatening condition for many. Traditionally, patients manage PAH with continuous infusion therapy, a demanding treatment requiring a portable pump. Introducing WINREVAIR™ as a novel therapy option, it stands to enhance disease management and significantly uplift patients' quality of life by offering an alternative to the relentless pump regimen.

"Currently, our most aggressively-treated patients are on oral medications in addition to lifelong infusion therapy. Sotatercept was studied added on to our current PAH medications. It is a less burdensome drug as compared to some of our other PAH therapies, in that it's a subcutaneous injection every three weeks," said Jean Elwing, MD, Professor of Internal Medicine in the UC College of Medicine's Division of Pulmonary, Critical Care and Sleep Medicine and Director of the Pulmonary Hypertension Program.

The Role of Background Therapy in PAH Management

The evolution of background PAH therapy has been instrumental in managing the disease, setting the stage for transformative treatments like WINREVAIR™. With the integration of sotatercept into the therapeutic arsenal, patients now have access to a broader spectrum of care options, addressing the need for a holistic approach to PAH management.

UC Health provides care for approximately 1,000 individuals facing pulmonary hypertension, half of whom are battling pulmonary arterial hypertension (PAH) specifically. In alignment with the latest advancements, the health system is poised to introduce WINREVAIR™ as a new treatment option for patients diagnosed with PAH, marking a significant step forward in patient care and treatment accessibility.

Addressing Pulmonary Vascular Resistance Through Innovative Therapy

The introduction of sotatercept marks a significant milestone in targeting pulmonary vascular resistance, a key factor in PAH progression. By focusing on the underlying mechanisms of the disease, researchers aim to offer new hope to patients facing the challenges of increased vascular resistance.

For over a decade, UC Health alongside the University of Cincinnati has spearheaded advancements in pulmonary arterial hypertension (PAH) treatment and research. Achieving recognition as one of the nation's initial five PAH-certified centers in 2014, they continue to play a pivotal role in the majority of PAH clinical research across the United States, further emphasizing their commitment to pioneering in the field.

Additionally, UC Health and the University of Cincinnati have contributed significantly to the research that led to WINREVAIR™'s FDA approval, playing key roles in crucial clinical trials such as the Phase 3 STELLAR trial and its subsequent open-label extension, SOTERIA. Their involvement underscores the institutions' pivotal contributions to advancing PAH therapy and their leadership in the field.

The STELLAR Trial showcased sotatercept's ability to amplify the effectiveness of existing PAH treatments, marking a notable improvement in the clinical landscape. This addition has been shown to significantly enhance patient outcomes, including the severity of hemodynamic disease, exercise capacity, overall symptoms, and quality of life for those with pulmonary arterial hypertension.

"Across the board, it seems to have decreased the burden of pulmonary vascular disease in the treated patients," Dr. Jose said.

Numerous PAH patients participating in the studies were at a critical juncture in their treatment journey, having already received the most comprehensive therapy options available. For many, the prospect of a lung transplant loomed as the subsequent step in their care.

UC Health and the University of Cincinnati continue their pivotal research efforts through participation in the HYPERION and ZENITH trials. These studies aim to deepen our comprehension of sotatercept's effectiveness across various stages of pulmonary arterial hypertension (PAH), potentially broadening the scope of treatment benefits for this challenging condition.

Collaboration with United Therapeutics and the American Lung Association

The journey to FDA approval for WINREVAIR™ has been supported by collaborations with key stakeholders in the PAH field, including United Therapeutics and the American Lung Association. These partnerships underscore the collective effort to combat this rare disease, emphasizing the importance of community and shared knowledge in advancing PAH care.

Public link

Please use the above public link if you want to share this noodl on another website.