Patients suffering from pulmonary arterial hypertension (PAH) can soon turn to a new medication targeting the root cause of the condition. On Tuesday, Merck’s sotatercept, marketed as Winrevair, received the green light from the U.S. Food and Drug Administration (FDA) to treat adults with the progressive and life-threatening lung condition.
Pulmonary arterial hypertension is a rare and rapidly progressive disease that causes the blood vessels in the lungs to narrow. As a result, blood pressure rises, harming the heart. The disease affects roughly 40,000 Americans—more of them women than men. The disease strains the heart, leading to limited physical activity, heart failure, and reduced life expectancy. The five-year mortality rate is 43 percent.
How It Works
Winrevair works by stopping and reversing the narrowing of the lung’s blood vessels. Multiple studies conducted by Dr. Humbert and his research team have shown that patients who take the drug experience statistically significant improvement in overall quality of life markers. For example, 29 percent of patients who received Winrevair improved from their baseline after 24 weeks compared to the 14 percent who received a placebo. Additionally, treatment with Winrevair showed an 84 percent reduction in the occurrence of death from any cause or clinical worsening in pulmonary arterial hypertension events compared to the placebo.“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,” Dr. Humbert said in the press release.
Furthermore, patients who took Winrevair showed a significant improvement in their ability to walk for six minutes compared to those who took a placebo. At Week 24, patients could walk 134 feet more in a six-minute walking test than those who took the placebo. In the trial, patients received stable background therapy.
Adverse Events
The medication is not without risks. At least 10 percent of patients reported adverse events, including nosebleeds, capillary dilation resulting in purple and blotchy skin, and dizziness. Severe bleeding occurred in 4 percent of the patients. Winrevair can also increase hemoglobin counts, leading to a condition known as erythrocytosis. Health care providers prescribing the medication should monitor hemoglobin and platelets after each dose for the first five doses or longer if values are unstable.
The medication may also impair male and female fertility, according to animal studies. It can also harm the fetus if a woman is pregnant.
Even with the warnings, the medical community finds the novel drug to be a boon to patients suffering from this rare and deadly disease.
“The Pulmonary Hypertension Association welcomes the development of new therapies for those with PAH,” Matt Granato, president and chief executive officer of the Pulmonary Hypertension Association, said in a news release. “A diagnosis of PAH is a life-changing experience for patients and families due to its chronic, progressive nature. 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.”
