Vertex Pharmaceuticals Inc., a biotechnology company looking to put more emphasis on drugs for the rare genetic disease cystic fibrosis, reported Thursday that a test of a CF drug called ivacaftor and marketed in the US under the name of Kalydeco “did not meet its primary endpoint.”
In early 2012, Kalydeco won approval from the Food and Drug Administration to market Kalydeco as a treatment for a subset of CF patients. The population of CF patients in the US, Europe, and Australia is estimated at 75,000. Nearly all eligible patients have started receiving treatment with Kalydeco in the US and Europe, and Vertex expects the medicine to generate $360 to $365 million in 2013.
In the study that Vertex reported on in Thursday’s press release, the company was looking to see if a wider segment of the CF population could benefit from the drug.
One positive takeaway from the study, Vertex said, is that Kalydeco/ ivacaftor showed significant benefits for patients 18 and older in which the disease is well established.
“While we are disappointed that the study in people with the R117H mutation did not meet its primary endpoint, we are encouraged by the significant improvements in lung function and other measures of CF observed in the subset of patients ages 18 and older who had established lung disease,” Robert Kauffman, MD, senior vice president and chief medical officer at Vertex, said in a statement. “We look forward to meeting with the FDA early next year to discuss these data with the goal of bringing ivacaftor to additional people with CF who may benefit from treatment.”
“With each study of ivacaftor, we continue to learn more about this disease and the effect of ivacaftor in patients with different CF mutations, ages and severity of disease,” he added.
In October, meanwhile, the company acknowledged it misjudged the market for its lead drug, a hepatitis C pill called Incivek. With the drug’s sales dropping sharply, Vertex said it was cutting about 17 percent of its total workforce, or 370 jobs — including 175 in Massachusetts — and would return $4.4 million in state tax incentives it received for promising to create jobs.
Vertex has just begun to move 1,300 employees from Cambridge into a new $800 million headquarters on the South Boston Waterfront. Most of those employees will be in the building by early January.
Chris Reidy can be reached at firstname.lastname@example.org.