FDA approves new combination pill for cystic fibrosis from Vertex Pharmaceuticals

Federal regulators on Thursday approved a new drug that treats the underlying cause of cystic fibrosis and eventually might be used to help almost half of the 30,000 patients in the United States with the fatal genetic disease.

Orkambi, a combination of Vertex’s approved drug Kalydeco and a new compound called lumacaftor, is intended for patients aged 12 and above, the USA Food and Drug Administration said on Thursday. The approval notice was posted to the agency’s website.

Orkambi and Kalydeco aim at fixing the underlying condition caused by the misbehaving gene, unlike current drugs which treat related symptoms.

With the agreement, Orkambi will certainly be now available to about 10,500 those in the United States, more often the two, 000 who might consider Kalydeco, which actually impacts on a special familial change that brings about cystic fibrosis.

In 2012, Vertex also won approval for the first drug aimed at counteracting the genetic defect behind cystic fibrosis, which causes the body to produce excess amounts of thick mucus that leads to loss of lung function and chronic infections.

©2015 by The Associated Press.

In the 1950s, children with cystic fibrosis seldom survived long enough to complete elementary school. Most patients die in their mid-20s.

Expectations for Vertex’s new drug have been tempered by study results that, while statistically significant, were not as dramatic as those first reported with Kalydeco. Kalydeco was developed for patients who have a problem with a protein on their cell walls, which does not properly balance the flow of water and salt from the cell.

Shares of Vertex rose $4.71, or 3.7 percent, to $130.90 before trading was halted at about midday. Vertex, which moved from Cambridge to a new $800 million campus on Boston’s Fan Pier last year, has become one of the largest Massachusetts biotech companies- with a stock market value of more than $30 billion- despite losing money for all but one of its 26 years.

Analysts have estimated the combination drug will generate billions of sales and profits for Vertex in the coming years- and more than $53 million for a dozen Vertex senior executives who were granted one-time bonuses that will be paid if the company tips into profitability.