New treatment for cystic fibrosis could benefit nearly half of patients

According to a study released by the New England Journal of Medicine, two large clinical trials have showed good results in treating people who suffered from cystic fibrosis.

It is the lung disease which affects tens of thousands of people in the United States. The trials were conducted on 1,108 cystic fibrosis patients from six countries between April 2013 and April 2014.

The results showed improved in lung function was and speed of pulmonary exacerbation was lowered as compared to placebos. The researchers also reported improvements in breathing and weight.

The report also claimed that the clinical benefit was sustained for the entire duration of the studies. Also, there were acceptable side-effect results of dose regimens.

Boston-based company Vertex cocktailed an existing drug Kalydeco with the compound Lumacaftor in order to create Orkambi. This drug aimed at prolonging the lives of children born with the genetic disorder.

Lumacaftor was not approved by the FDA. Kalydeco is known genetically as ivacaftor and costs about $250,000 per patient per year in the US.

Orkambi was created to treat nearly half of the 30,000 cystic fibrosis patients in the country. According to some estimates, it could cost up to $50,000 a year more than its predecessor.

If Orkambi is approved, it would be the first medicine to treat patients aged 12 or older with the most common form of cystic fibrosis, known as F508del. Researchers hope it will also increase the survival rate.

The median life expectancy is 37 years in the US. However, it is much lower in less developed countries. Many people die before the age of 40 due to years of overproduced thick mucus, which leaves 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.