Results of two large clinical trials showed major promise in treating people suffering from cystic fibrosis, the lung disease affecting tens of thousands of people in the United States.
Controlled trials - which tested 1,108 cystic fibrosis patients from six countries between April 2013 and April 2014 - improved overall lung function and lowered the speed of pulmonary exacerbation as compared to placebos, according to a study released by the New England Journal of Medicine. Improvements in breathing and weight, as well as fewer hospital visits, were also recorded.
"The clinical benefit was sustained for the entire duration of the studies," read the Journal, adding that both dose regimens had acceptable side-effect results.
Boston-based company Vertex cocktailed existing drug Kalydeco with the compound Lumacaftor to create Orkambi, the "breakthrough" drug aimed at prolonging the lives of children born with the genetic disorder. Lumacaftor is not approved by the Food and Drug Administration, but Kalydeco - known genetically as ivacaftor -costs about $250,000 per patient per year in the U.S.
Orkambi is intended to treat nearly half of the 30,000 cystic fibrosis patients in the country. It could cost up to $50,000 a year more than its predecessor, according to some estimates.
If approved, Orkambi would be the first medicine to treat patients aged 12 or older with the most common form of cystic fibrosis, known as F508del. While the trials only showed to reduce the disease's severity, researchers hope it will increase the survival rate.
The median life expectancy is 37-years-old in the U.S. but much lower in less developed countries. Most die before the age of 40 because years of overproduced thick mucus leave the lungs clogged, damaged, and vulnerable to infection.
A federal advisory committee approved recommending Orkambi to the FDA by a 12-to-1 vote. A final decision is expected by July 5.
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