Cystic Fibrosis

MPR Weekly Dose Podcast: Episode 10

This week we talk about the resurrection of Biogen’s novel Alzheimer treatment; A “landmark” approval in cystic fibrosis; Expanded indication for Farxiga; The first topical minocycline formulation for acne vulgaris; and the Johnson & Johnson baby powder recall.

FDA Approves Triple Combination Therapy for Cystic Fibrosis

The Food and Drug Administration (FDA) has approved Trikafta (elexacaftor/tezacaftor/ivacaftor; Vertex) for the treatment of patients aged 12 years and older with cystic fibrosis (CF) who have ≥1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Trikafta is a fixed-dose combination containing elexacaftor 100mg, tezacaftor 50mg, and ivacaftor 75mg that is co-packaged…

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…