FDA approves new drug that may help stop and even reverse a rare, fatal

CNN
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When doctors told Katrina Barry that she had a rare and serious condition called pulmonary arterial hypertension or PAH, they warned her not to Google it.

Come on, she thought; they wanted a young woman who was bound for graduate school, who had survived a transatlantic plane flight while having a heart attack and now open-heart surgery, not to look up the condition that kept trying to kill her?

Waiting for her on the internet was some chilling information. PAH affects about 500 to 1,000 Americans each year, often women between the ages of 30 and 60, according to the American Lung Association.

Barry, who was 25 at the time, learned from her reading that she had two to five years to live, based on how severe her condition already was.

Then her medical team offered her a potential lifeline: a first-of-its-kind experimental drug called sotatercept that corrals a growth factor that is overproduced by people with PAH, potentially changing the underlying biology of the disease.

She signed up for a study to test the medication, given every three weeks by injection under the skin.

She started treatment at the dawn of the Covid-19 pandemic, in March 2020. Barry said she diligently wore an N95 mask and two layers of clothing and scrubbed herself down after every trip to the hospital for her injections to avoid getting Covid along the way.

“I had to go to the hospital because I was dying if I didn’t get this drug,” she said. “This was the only chance I had at survival.”

On Tuesday, the US Food and Drug Administration approved the medication that Barry has taken for four years, a drug she credits with keeping her alive and allowing her to resume many of the activities she enjoyed before her diagnosis.

Merck, the company that manufactures the drug, said it will be sold under the brand name Winrevair.

Barry, now 29, is a patient consultant for Merck but said she has not received compensation for telling her story.

Winrevair is a biologic drug designed to grab onto and trap proteins called activins that are overproduced in PAH. These proteins cause the walls of a person’s blood vessels to keep growing and thicken over time.

As the blood vessels narrow, the heart is forced to work harder to pump blood to the lungs. This stress eventually damages the heart, causing it to fail.

Without treatment, people who have PAH usually live about two to three years after their diagnosis. Treatment with a combination of drugs that dilate, or relax, blood vessels can improve this outlook, but they are not a cure.

“This medication has a much different mechanism of action, and it may actually rebalance some of the growth that goes on in … the very small pulmonary arterioles where the disease starts,” said Dr. Vallerie McLaughlin, a cardiologist at the University of Michigan who helped study sotatercept.

“That may attack the disease from a different angle and allow for what we returned as reverse remodeling,” she said, meaning patients could actually improve over time while on the medication.

Winrevair is the first new type of drug to come along for patients with PAH in a long while, said Dr. Panagis Galiatsatos, a pulmonary and critical care medicine specialist at Johns Hopkins Bayview Medical Center and a spokesperson for the American Lung Association.

In animal studies conducted before the human trials, the drug looked like it could do more than just treat symptoms: It seemed like it might be able to stop the thickening of the blood vessels and perhaps prolong patients’ lives, but those benefits have not been proven in humans.

Instead, Galiatsatos said, the FDA approved the drug based on other signs of effectiveness,…