Vertex CF drug disappoints

Vertex Pharmaceuticals Inc., which is looking to put more emphasis on developing drugs for cystic fibrosis, reported Thursday that a test of a CF drug called ivacaftor (marketed in the United States as Kalydeco) “did not meet its primary endpoint.”

In 2012, Kalydeco won Food and Drug Administration approval as a treatment for a subset of CF patients.

The population of CF patients in the United States, Europe, and Australia is estimated at just 75,000. Nearly all eligible patients have started receiving treatment with Kalydeco in the United States and Europe.


In the study Vertex reported on Thursday, the company was looking to see if a wider segment of the CF population could benefit from the drug.

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One positive takeaway from the study, Vertex said, is that Kalydeco showed significant benefits for patients 18 and older in whom 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,” Dr. Robert Kauffman,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.”

In October, the company acknowledged it had misjudged the market for its lead drug, a hepatitis C pill called Incivek. With that drug’s sales dropping sharply, Vertex said it was cutting about 17 percent of its workforce, or 370 jobs — including 175 in Massachusetts — and returning $4.4 million in state tax incentives it had 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.

Chris Reidy