Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Receives the US FDA’s Approval to Treat the Underlying Cause of Cystic Fibrosis

Shots:

• The approval is based on two global P-III studies assessing Trikafta in 403 & 107 patients with CF aged ≥12yrs. with at least one F508del mutation and one minimal function mutation (F/MF) & in people with two F508del mutations (F/F) for 24wks. & 4wks. respectively
• The two P-II studies result demonstrated improvement in FEV₁ and respiratory symptoms in F/MF study @24wks. with a reduction in the rate of pulmonary exacerbations and improvements in BMI
• Trikafta is a prescription therapy to treat CF, currently being evaluated in P-III study in patients aged 6-11yrs with F/MF and F/F CF mutations and will be evaluated in children aged <6yrs. Vertex has submitted EMA’s MAA for the combination regimen

Click here to­ read full press release/ article | Ref: Vertex | Image: Wbur