Cystic Fibrosis Archives - Page 2 of 3 - MPR

Cystic Fibrosis

FDA to Review Elexacaftor, Tezacaftor, Ivacaftor Combo for Cystic Fibrosis

The Food and Drug Administration (FDA) has accepted for priority review the New Drug Application (NDA) for elexacaftor (VX-445), tezacaftor and ivacaftor triple combination regimen for patients with cystic fibrosis. Elexacaftor is an investigational therapy, while the combination of tezacaftor and ivacaftor and ivacaftor alone are both already approved treatments for cystic fibrosis. The NDA…

Novel Triple Combination Therapy for Cystic Fibrosis Submitted for FDA Review

A New Drug Application (NDA) for the investigational therapy elexacaftor (VX-445), for use in combination with tezacaftor and ivacaftor in the treatment of cystic fibrosis, has been submitted to the FDA by Vertex Pharmaceuticals.  Elexacaftor and tezacaftor facilitate the cellular processing and trafficking of normal and select mutant forms of cystic fibrosis transmembrane conductance regulator…

Symdeko Approved to Treat Younger Cystic Fibrosis Patients

The Food and Drug Administration (FDA) has approved Symdeko (tezacaftor/ivacaftor and ivacaftor; Vertex Pharmaceuticals) for the treatment of patients aged ≥6 years with cystic fibrosis (CF) who are homozygous for the F508del mutation or who have at least 1 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor. Previously,…