FDA approves new drug that may help stop and even reverse a rare, fatal condition that doctors call a ‘ticking time bomb’ – CNN

<h2>FDA approves new drug that may help stop and even reverse a rare, fatal condition that doctors call a ‘ticking time bomb’ – CNN</h2><div class="wp-block-image"><figure class="alignright size-large" style="padding:0px 20px;"><img height="100px" src="https://images.unsplash.com/photo-1605108083603-85696109df99?crop=entropy&cs=srgb&fm=jpg&ixid=M3w2MDA1ODl8MHwxfHNlYXJjaHwxfHxhcHByb3ZlcyUyMG5ld3xlbnwwfHx8fDE3MTcyNjQ4MDN8MA&ixlib=rb-4.0.3&q=85" alt=""/></figure></div><p>CNN —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 was approved by the FDA on Tuesday to help treat pulmonary arterial hypertension. Courtesy MerckWinrevair 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, including whether patients were able to walk farther in six minutes than they could before they started taking the drug, a clinical measure called the six-minute walk test.In a study that included 323 people who took either sotatercept or a placebo for six months, those who got the drug were able to walk 34 meters – about half a city block – farther in six minutes than they could before they started treatment, while people in the placebo group were about to walk only about 1 meter more than they could when they started the study. Both groups were also taking the standard medications for the condition, which help relax blood vessels to improve blood flow.By the end of the study, nine participants, or about 5%, in the sotatercept group had died or had their disease worsen, while 42 patients, or about 26%, in the group taking a placebo had died or gotten worse.The FDA was willing to act fast to approve the medication because the disease, Galiatsatos said, is a “ticking time bomb” for people who don’t have time to wait for longer studies.The study that led to the drug’s approval was published in the New England Journal of Medicine last year.“Many of</p>