On July 5, the FDA will announce whether or not the new drug meant to treat people diagnosed with cystic fibrosis has been approved for US usage.
The drug, developed by Vertex, will be named Orkambi and will consist of two active substances, one of them is Ivacaftor, which is already being used to treat patients that have a rare genetic mutations, which affects about 4% of those with cystic fibrosis.
The other drug has not yet been approved, and it is called Lumacaftor. Lumacaftor is being developed by Vertex, the same company which aims at getting Orkambi approved.
The utility of these drugs used as a combo was revealed by the clinical trials published in the New England Journal of Medicine.
In the trials, participated around 1000 people of age 12 or older and all those who received the drug, showed significant improvement in their breathing, as well as other aspects of their life.
And if this drug is as good as it promises to be, soon we will be able to see cystic fibrosis patients live longer, healthier lives.
The new drug could be of great use to about 15,000 persons diagnosed with the disease in the US alone, but statistics reveal that there are over 75,000 people suffering from cystic fibrosis in Europe, Australia and North America.
Cystic fibrosis is a genetic disorder that affects many organs of the human body like the pancreas, the intestine, the liver and the kidneys, but is particularly harsh on the lungs.
The disease is caused by the expression of two copies of the same gene for the protein CFTR, but there are also people who carry this genetic mutation and who are completely normal, because one of the two copies of the gene is not functional. These people are called carriers.
The disease itself consists of an overproduction of mucus which builds up and can cause infections and breathing problems in the lungs, the mucus can build up in the pancreas and prevent it from releasing its enzymes into the intestine etc.
Although new drugs are developed every year, currently there is no drug that can cure this genetic disease, but rather medicine that can help alleviate symptoms and extend the life expectancy of those suffering from cystic fibrosis.
Patients diagnosed with this disease have a life expectancy of 37 years, but thanks to the new Orkambi drug, this life expectancy might increase and there is a great hope that for many of them symptoms will improve.
Image Source: nytimes