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Vertex gets approval to treat toddlers with cystic fibrosis drug

The Food and Drug Administration on Tuesday approved the expanded use of Orkambi.
The Food and Drug Administration on Tuesday approved the expanded use of Orkambi.(Vertex Pharmaceuticals)

About 1,300 children ages 2 to 5 with cystic fibrosis in the US will be eligible to take a drug that Boston-based Vertex Pharmaceuticals Inc. already markets for older patients, as a result of a government ruling.

The Food and Drug Administration on Tuesday approved the expanded use of Orkambi after finding that it was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients 6 years old and older.

“For the first time, children ages 2 through 5 who have the most common form of CF have a treatment for the underlying cause of their disease,” said Dr. Reshma Kewalramani, executive vice president and chief medical offier of Vertex.

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Orkambi was already approved in the US for the treatment of cystic fibrosis in patients ages 6 and older who have two copies of a particular genetic mutation. But it needed to be test in kids younger than that with the same mutation.

The drug doesn’t come cheap. It now costs $272,000 a year per patient.

Cystic fibrosis is a rare obstructive lung disease that afflicts about 75,000 people in North America, Europe, and Australia.


Jonathan Saltzman can be reached at jsaltzman@globe.com