A young patient holds Kalydeco, a cystic-fibrosis treatment from Vertex just approved by the FDA. Certain cystic-fibrosis patients will now have an FDA-approved treatment that targets an underlying cause of their disease. But that drug — Kalydeco, from Vertex Pharmaceuticals — won’t come cheap. As Dow Jones Newswires reports , the annual cost will be $294,000. Vertex has set up a patient-assistance program to help patients pay for the treatment. Kalydeco was approved to treat the estimated 4% of cystic-fibrosis patients who have a mutation called G551D in a certain gene responsible for the disease. About 1,200 people in the U.S. have that mutation, DJN says. Kalydeco was approved for use only in patients aged six and up; Vertex is planning to study the drug in younger children. The company is also studying the drug in cystic-fibrosis patients with different mutations and in combination with other medications. As the WSJ reported in the fall, progress in drug development for cystic fibrosis has come more than 20 years after the gene responsible for the disease was identified, in 1989. Pinpointing the gene was only one step. Scientists had to come up with a hypothesis

as to how the defective gene actually causes the disease, then devise a way to address the problem. As the paper reported: The gene makes the channels that carry chloride in cells up to airways, where it’s needed for clearing mucus that builds up, some scientists believe. Mutations impede the flow of chloride, causing the mucus to accumulate and impeding the hair-like particles called cilia from beating back and forth to clear out the mucus. Attempts to use gene therapy to replace the mutated gene didn’t pan out, so researchers have instead turned to drugs to help chloride to reach the airways. Other companies, including PTC Therapeutics, have experimental therapies at various stages of the development pipeline. The median lifespan for cystic-fibrosis patients has risen over the years thanks to aggressive treatments addressing the disease’s symptoms. But most will still not live beyond their 30s and 40s. Bonus: Not Just a Cheerleader: Foundation Helped Drive Cystic-Fibrosis Research Correction: An earlier version of this post gave the incorrect number of U.S. patients with the mutation. Image: Brett Deering for the WSJ

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FDA Approves Vertex’s Kalydeco, But It Won’t Come Cheap