The US Federal Drug Authority has approved a new drug that will help people with cystic fibrosis. Cystic Fibrosis affects 30000 people in the US as per the figures released by US FDA. Cystic fibrosis is a debilitating genetic disorder and is characterized by the formation of viscous mucus that builds in the lungs, digestive tract and different parts of the body.
The disease leads to severe respiratory and digestive problems including complications like infections and diabetes which further aggravates the condition. The buildup of mucous makes breathing an effort.
As per the Cystic Fibrosis Foundation, the new medication Orkambi can help people suffering from Cystic Fibrosis who are 12 years or above in age and bear two copies of the F508del gene mutation that is the most common type of CF mutation. Orkambi was officially approved by the FDA on July 2. According to the CF Foundation Orkambi marks one more step towards the development of drugs that are meant to allay the symptoms of the disease by targeting the cause of CF.
The approval of the drug by FDA has been welcomed by Robert Beall, President and CEO of the Cystic Fibrosis Foundation who said that the approval marks a major milestone in what was a long and arduous journey to discover and develop drugs that will treat the cause of CF. More than 1000 volunteers suffering from CF had participated in the clinical trials that culminated with the approval of Orkambi by the FDA.
Orkambi becomes the second drug that has been approved by FDA from the CF Foundations research program in association with Vertex Pharmaceuticals. Earlier, ivacaftor was approved by FDA for treating people who have certain rare CF mutations. Orkambi’s approval will benefit more than 10000 people who are living with Cystic Fibrosis, and the drug treats the underlying genetic cause of the disease.