Vertex Wins U.S. Approval for Cystic Fibrosis Combo Orkambi

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Vertex Pharmaceuticals Inc. won U.S. approval for its combination treatment for the most common form of cystic fibrosis, giving the drugmaker what analysts have projected will be a new blockbuster.

The Food and Drug Administration cleared the medicine, called Orkambi, for sale for patients 12 and older who suffer from the deadly lung disease, according to a letter posted in an FDA database. Orkambi is a combination of a drug called Kalydeco that Vertex already sells for a smaller group of cystic fibrosis patients and another medicine called lumacaftor.

Vertex will charge $259,000 a year for Orkambi, the company said on a conference call Thursday. Kalydeco cost $294,000 a year when it came to market in 2012 and last year had sales of $464 million. Revenue from Orkambi could reach $3.42 billion in 2018, analysts estimate.

“Today’s approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic fibrosis,” John Jenkins, director of the FDA’s office of new drugs, said in a statement.

Orkambi should be available within days, Vertex said. The shares rose 4 percent to $131.26 at the close in New York.

The FDA designated Orkambi an orphan drug, which means it will qualify for seven years of marketing exclusivity free from generic competition, since it treats a rare disease. Vertex studied Orkambi in two clinical trials that showed patients who took the drug, given as two pills every 12 hours, experienced improved lung function compared to those who took a placebo, the FDA said.

Combination Treatment

The FDA originally approved Kalydeco for use in about 1,200 U.S. cystic fibrosis patients who have a certain genetic mutation. Since then, the drug’s use has been expanded to about 2,000 people. By combining Kalydeco with lumacaftor, Boston-based Vertex is seeking to treat an additional 8,500 patients.

Cystic fibrosis affects 30,000 children and adults in the U.S., according to the Cystic Fibrosis Foundation. It can be a fatal disease that causes patients’ lungs and other organs to clog with thick, sticky mucus.

Orkambi is targeted at patients with two copies of the F508del mutation in the CFTR gene, defects in which lead to cystic fibrosis. About 50 percent of people with the condition in the U.S. have two copies of the F508del mutation, according to the Cystic Fibrosis Foundation.