On Tuesday, the Food and Drug Administration approved a drug from… Merck It's designed to treat a progressive, life-threatening lung condition, representing a win for both the drugmaker and patients with the rare disease.
The agency has given the green light to the treatment, to be marketed as Winrevair, for adults with pulmonary arterial hypertension. This decision is a big step for the approximately 40,000 people in the United States living with the disease, because Winrevair is the first drug to target the root cause of the condition. Other medications available only help manage symptoms.
The condition refers to the narrowing of small blood vessels in the lungs. This leads to high blood pressure in the arteries that carry blood from the heart to the lungs, which can damage the heart and lead to limited physical activity. From diagnosis, the mortality rate for patients is 43% within five years, according to Merck.
Merck estimates that Winrevair will be available in select specialty pharmacies in the United States by the end of April, according to a company statement. The medication is an injection that is taken every three weeks and is distributed in a single vial or a double vial.
A Merck spokesman said in a statement that its price will be $14,000 per vial before insurance. But the company has a program that offers eligible patients help with direct costs and co-payments.
Winrevair is meant to be used in conjunction with current treatments for this condition to increase exercise capacity, reduce the severity of PAH, and reduce the risk of disease worsening.
The approval is crucial for Merck, which is working to diversify its revenue stream with its top-selling cancer immunotherapy Keytruda on the verge of losing market exclusivity in 2028.
In a note this month, JPMorgan analyst Chris Schott estimated that Winrevair would reach global annual sales of about $5 billion by 2030 and emerge as one of Merck's “biggest growth engines.”
“This is a really great opportunity for the company, but more importantly, it's a very important opportunity for patients,” Eliav Bar, Merck's chief medical officer, told CNBC. He pointed out that the drug would be a “paradigm shift” for patients suffering from pulmonary arterial hypertension.
The company acquired the rights to Winrevair through its $11.5 billion acquisition of Acceleron Pharma in 2021. At the time, Merck estimated that PAH would be a roughly $7.5 billion market by 2026.
The FDA approval is based on data from a late-stage trial, which followed more than 300 patients with moderate-stage pulmonary arterial hypertension who were already taking another medication for the vascular condition.
The study found that Winrevair combined with the existing treatment helped patients with the condition walk about 40.8 meters in six minutes more than those who received a placebo, after 24 weeks of the trial.
“There is a huge improvement in people's ability to exercise and move around,” Barr said. “Because this disease makes people very confined to their homes. They suffer from shortness of breath and cannot move.”
Winrevair combined with an existing drug also significantly improved eight of nine secondary objectives in the study. This includes an 84% reduction in the risk of death or worsening of the condition compared to the drug alone.
Severe and serious adverse events were less common in the group of patients who took Winrevair compared to those who received a placebo, according to the trial. Side effects that occurred frequently included nosebleeds, headache, and rash, among others.
One notable advantage of Winrevair is that patients or caregivers can inject it subcutaneously with appropriate training from the healthcare provider. Meanwhile, some current treatments for PAH must be administered by medical professionals in an infusion center
“One of the things we heard very loud and clear, from both patients and doctors, is that they want something you can get at home,” Barr said.
Merck is continuing to study Winrevair in other Phase 2 and Phase 3 trials.
These trials include late-stage studies in patients with more advanced PAH disease and those in the first year after diagnosis. Merck said it expects these trials to end around 2025 and 2026
