WSJ July 06, 2015
The U.S. Food and Drug Administration approved Vertex Pharmaceuticals Inc.’s cystic-fibrosis drug Orkambi, which could treat as many as 8,500 patients in the U. S.—but at a whopping annual wholesale cost of $259,000 per patient.

The drug, a twice-daily oral tablet for patients 12 years and older, expands the company’s reach in the treatment of cystic fibrosis, the progressive lung disease that often kills patients in their 20s. Vertex said the new drug would be available within days. Orkambi is aimed at treating patients with a genetic mutation that is the leading cause of cystic fibrosis.

The U.S. Food and Drug Administration approved Orkambi Thursday. The Vertex pill, taken twice per day, is targeted at a genetic defect found in almost half of cystic fibrosis patients. Orkambi sales are expected to transform Vertex into a sustainably profitable company for the first time since its founding in 1989.

In approving Orkambi, the FDA concurred with the positive recommendation from a panel of outside experts who voted 12-1 in May that two large clinical trials conducted by Vertex provided sufficient evidence to support the efficacy and safety of the drug. European drug regulators are also reviewing Orkambi with an approval decision expected in the fourth quarter.