FDA gives a nod to a new drug to treat cystic fibrosis

The US Food and Drug Administration have approved a new drug for the treatment of cystic fibrosis. The first of the triple-combination therapy which is available for the treatment of a common type of mutation of cystic fibrosis is Trikafta (tezacaftor/ivacaftor/elexacaftor) and its cost is around $311,000 in a year.

Patients who are above 12 years old and have at least a single F508del mutation in their CFTR (cystic fibrosis transmembrane conductance regulator) gene can be given Trikafta. As per FDA this accounts for nearly 90% of the patients of cystic fibrosis which is about nearly 27,000 people in US. Among the known 2000 CFTR gene mutations, F508del is the most common one. A particular gene mutation which results in a defective protein causes cystic fibrosis. A few people can be treated with the help of current drugs in which the defective protein is targeted however there are other people in whom the mutation does not respond to the treatments.

Trikafta is a combination of 3 drugs and is produced by Vertex Pharmaceuticals Inc. The defective protein that is produced due to CFTR mutation is made to function better by the use of this drug. Trikafta got the approval following 2 clinical trials which involved 510 patients. The assessment of a lung function measure was done in the trials known as ppFEV1 (percent predicted forced expiratory volume in one second). It measures the amount of air that can be exhaled by a person during forced breath. In one of the trials there was a 13.8% improvement in the average ppFEV1 value in comparison to those who had taken placebo. There was a 10% increase in ppFEV1 value due to the use of the drugs in the second trials compared to the use of ivacaftor/tezacaftor.

Dr. Ned Sharpless, acting Commissioner of FDA said that the approval was the result of the efforts for speeding up development of novel therapies for various complex diseases. He said that the approval of Trikafta has made available to most patients of cystic fibrosis a new treatment method especially the adolescents who did not have other options previously.

Cystic fibrosis is a life-threatening progressive disease which results in the formation of a thick mucus to build up in lungs, digestive tract or other body parts, which can result in serious digestive and respiratory problems as well as other complications like diabetes and infections.