Cystic Fibrosis Drug Wins Approval of F.D.A. Advisory Panel By ANDREW POLLACK MAY 12, 2015 A federal advisory committee on Tuesday recommended approval of a drug from Vertex Pharmaceuticals that might eventually help nearly half of patients with cystic fibrosis. The advisory committee to the Food and Drug Administration voted 12 to 1 that the drug, which Vertex plans to call Orkambi, was safe and effective enough to be approved. The F.D.A. is supposed to decide by July 5. The drug would be the second cystic fibrosis drug for Vertex and the second in the world that works directly to counteract the genetic defect that causes the disease, rather than just treat the symptoms. Vertex hopes the drug will propel the company to consistent profitability after 26 years in business. Vertex already sells Kalydeco, the first drug that works on the underlying genetic defect of cystic fibrosis, but it is applicable only to about 2,000 of the 30,000 cystic fibrosis patients in the United States. A scientist running tests at the Vertex site in San Diego. VERTEX The new drug, Orkambi, is intended for patients who have two copies of the most common mutation, known as F508del. About 14,000 patients are in this category, though the drug would be approved initially only for the approximately 8,500 of them who are at least 12 years old. Vertex is now testing the drug in children as young as 6. Geoff Porges, an analyst at Sanford C. Bernstein, estimates Orkambi could achieve $2.2 billion in annual sales in 2018 and would help Vertex achieve overall revenue of about $5 billion. Cystic fibrosis is an inherited disease caused by mutations in a gene. Sticky mucus builds up in the lungs, causing a gradual decline in the ability to breathe. Death from respiratory failure often occurs when people are in their 20s, 30s or 40s. An issue for the advisory committee was that Orkambi had what the F.D.A. said was only modest effectiveness, improving lung function by only about 3 percentage points relative to placebo. Some family members or advocates, some of them crying, pleaded with the committee to endorse the drug. Some patients who took the drug in clinical trials said it had made a huge difference in their lives, reducing their coughing, allowing them to exercise better, helping them gain weight or reducing how often they ended up in the hospital. “While on paper, Orkambi may not have given me drastic improvement in lung function, I’m here to tell you under no uncertain terms this drug has saved my life,” said Jeff Masters, 36, of Ann Arbor, Mich. He said Orkambi “brought me the best quality of life that I can remember,” even allowing him to run five-kilometer races. “A ‘no’ vote will throw me back into the prison of cystic fibrosis,” he said. Orkambi is a combination of ivacaftor, the ingredient in Kalydeco, and a second drug, lumacaftor. The F.D.A. staff suggested that Orkambi’s effect on lung function did not seem that much greater than that of ivacaftor alone for the patients in question, raising questions about whether the second drug, lumacaftor, really added anything. Typically, to be approved, a combination drug like Orkambi must be shown to be better than either component alone. But Vertex pointed out that the F.D.A. had agreed to trials that did not compare Orkambi to its components, only to a placebo, for good reason: Earlier studies had suggested the two components individually did not work. But the study of ivacaftor alone left some doubt to how effective it might be. The committee, meeting in Gaithersburg, Md., was split on whether lumacaftor really added to the efficacy; many members said that question could not be answered from the available data. But most of them said that in the end that did not really matter. The combination drug worked and there did not appear to be any real safety concerns with the combination compared with ivacaftor alone. “I felt that given where we are today, I decided to err on the side of giving patients access to medicine, given the tremendous unmet medical need,” said a committee member, Elaine H. Morrato, associate professor of health systems, management and policy at the Colorado School of Public Health. If the drug is approved, Vertex could still face challenges getting insurers to pay for it, given the somewhat questionable efficacy. Kalydeco costs more than $300,000 a year, although Orkambi is expected to be a little less expensive, because it is for a larger patient population and is not as effective. Michael Yee of RBC Capital Markets, for instance, expects the price will be $225,000 to $250,000 a year.Chcesz być na bieżąco z nowościami, które mogę Cię zainteresować, zapisz się do naszego newslettera[wysija_form id=”1″]