Explore the innovative pulmonary arterial hypertension treatment sotatercept, approved by the Food and Drug Administration after rigorous clinical trials.

Story by: David Steen Martin; Reviewed by John W. McConnell, M.D. on May 15, 2024

A new pulmonary arterial hypertension treatment has been approved by the Food and Drug Administration (FDA) after completing clinical trials at Norton Pulmonary Specialists and other leading pulmonary hypertension treatment centers around the world.

Sotatercept, sold under the brand name Winrevair, has been shown to increase exercise capacity, reduce symptoms that affect day-to-day activities and lower the risk of clinical worsening from the condition.

The FDA approved sotatercept in March under its expedited approval process after determining sotatercept was a “breakthrough drug” that could be a significantly better treatment for a life-threatening condition such as pulmonary arterial hypertension. The condition’s name is sometimes shortened to PAH.

“There’s no cure for pulmonary arterial hypertension, which is why new and better therapies like sotatercept are so important for PAH patients” said John W. McConnell, M.D., a principal investigator in the clinical trial that led to sotatercept’s approval. Dr. McConnell is a pulmonologist with Norton Pulmonary Specialists and medical director of Norton Healthcare’s pulmonary hypertension program.

As a principal investigator in the recently concluded sotatercept clinical trial, Dr. McConnell has extensive experience treating patients with the drug. He is also the principal investigator in two additional studies of sotatercept in combination with other medicines for pulmonary arterial hypertension.

Pulmonary arterial hypertension is rare condition that gets worse over time and is ultimately life-threatening. The condition can lead to right heart failure and can be deadly after a few years.

Pulmonary hypertension is a broad term for high blood pressure that’s localized in the lungs, unlike more widespread hypertension, which is high blood pressure throughout the body. Pulmonary arterial hypertension is the term used to describe rarer instances when the condition is the result of narrowed or blocked pulmonary arteries.

Pulmonary arterial hypertension results from the excessive growth and dysfunction of cells that line the arteries carrying blood from the heart to the lungs. Smaller arteries in the lungs thicken and narrow, restricting blood flow and contributing to high blood pressure in the lung that can damage the heart and restrict physical activity. A right heart catheterization measures pulmonary artery pressure and is a critical part of an accurate diagnosis.

Sotatercept works by inhibiting the cell overproduction and allowing the pulmonary artery and smaller arteries to revert to a healthier structure. It’s the first drug to do this.

Pulmonary arterial hypertension is most common in women between the ages of 30 and 60, according to the American Lung Association. Because the cause of pulmonary arterial hypertension isn’t known, it is sometimes called idiopathic pulmonary arterial hypertension. The condition may have a genetic component, and some conditions increase risk for the disease. Congenital heart disease is a risk factor for the disease, according to the American Heart Association, along with  connective tissue disease, including scleroderma and lupus.

Sotatercept is delivered by an injection that the patient can administer every three weeks. The medication is expensive. Even with insurance, some pulmonary arterial hypertension patients need help with the copay, according to Dr. McConnell. Nurses at Norton Pulmonary Specialists are available to help patients with the process of signing up for the drug once it’s prescribed and applying to sources of copay assistance, according to Dr. McConnell.

Pulmonary arterial hypertension affects an estimated 40,000 people in the United States. As many as half of them may be eligible for sotatercept. Shortness of breath during exercise is a symptom of pulmonary arterial hypertension, according to the National Heart, Lung, and Blood Institute. Symptoms tend to get worse over time and may include dizziness, swelling of the ankles or legs, chest pain, and a racing pulse.

By making the heart work harder to pump blood through the lungs, pulmonary arterial hypertension puts a significant strain on the heart. Over time, the heart muscle becomes weak from the extra effort, leading to heart failure.

Pulmonary arterial hypertension also affects quality of life. Many people with pulmonary arterial hypertension need a continuous infusion therapy administered by a pump that they must carry with them at all times. Oxygen therapy delivered by a constantly present tank may be required as disease progresses.

Clinical trial findings

The clinical trial compared patients who received the new therapy with those who received a placebo.

Among the findings, treatment with sotatercept:

• Resulted in an improvement in day-to-day function for almost a third of people taking the drug, double the number of those who improved taking the placebo
• Led to bigger improvements in six-minute walking distance
• Made it easier for the heart to pump blood through the blood vessels in the lungs
• Reduced the chances of worsening events or death by 84%

Serious side effects were rare, but sotatercept did increase the chance of serious bleeding. This was more likely in patients who also were receiving prostacyclin infusion, were on blood thinners or had low platelet counts. Sotatercept also may increase hemoglobin, which could lead to erythrocytosis, a thickening of the blood that makes it less able to travel through blood vessels and organs.

The most recently completed clinical trial tested the drug in patients who met the World Health Organization criteria for functional class II or III, meaning they had no symptoms while resting, but had varying severity of symptoms while physically active.

A study currently underway at Norton Pulmonary Specialists is testing the safety and efficacy of the drug in patients who are in class III or IV and at high risk of death.

Sotatercept is given along with conventional background medication for pulmonary arterial hypertension. Lung transplant is also a treatment for advanced stages of the disease.

Current clinical trials for pulmonary arterial hypertension at Norton Pulmonary Specialists