The US FDA today has given its
nod to Trikafta (elexacaftor/ivacaftor/tezacaftor) of Vertex Pharmaceuticals, the
first of its kind triple combination therapy for the patients with cystic fibrosis.
The drug, Trikafta, has been
approved for patients of age 12 years and older who have at least one F508del
mutation in CFTR (cystic fibrosis transmembrane conductance regulator) gene, which
mostly affects 90% of all the cystic fibrosis population.
Cystic fibrosis can be a result of more than 1000 possible mutations in the CFTR gene that hampers the pathway of chloride channels which disrupts the traverse of chlorine ions and water molecules across cell membranes. The...
