ivacaftor

Cystic Fibrosis: Causes and Treatment

Symptoms In cystic fibrosis (CF, cystic fibrosis), different organ systems are affected, resulting in a heterogeneous clinical picture with symptoms of varying severity: Lower respiratory tract: Chronic cough with viscous mucus formation, obstruction, recurrent infectious diseases, e.g., with , inflammation, remodeling of the lungs (fibrosis), pneumothorax, respiratory insufficiency, shortness of breath, wheezing, oxygen deficiency. Upper … Cystic Fibrosis: Causes and Treatment

Elexacaftor

Products Elexacaftor was approved in the United States in 2019 and in many countries in 2020 as a fixed-dose combination with tezacaftor and ivacaftor in film-coated tablet form (Trikafta). In the morning dose, all three active ingredients are included. In the evening dose, only ivacaftor. Structure and properties Elexacaftor (C26H34F3N7O4S, Mr = 597.7 g/mol) exists … Elexacaftor

Ivacaftor

Products Ivacaftor was approved by the FDA and EMA in 2012 and by Swissmedic in 2014 in film-coated tablet form (Kalydeco). Also available is a fixed combination with lumacaftor (Orkambi). In 2016, a granule was also approved. In 2018, the combination with tezacaftor was approved in the US and EU (Symdeko, Symkevi). In 2020, a … Ivacaftor

Lumacaftor

Products Lumacaftor is commercially available in a fixed combination with the CFTR potentiator ivacaftor in the form of film-coated tablets (Orkambi). The drug was approved in the EU and US in 2015 and in many countries in 2016. Structure and properties Lumacaftor (C24H18F2N2O5, Mr = 452.4 g/mol) exists as a white powder that is practically … Lumacaftor

Tezacaftor

Products Tezacaftor was approved in the US and EU in 2018 and in many countries in 2019 in fixed combination with previously registered ivacaftor in film-coated tablet form (Symdeko). In 2020, a fixed combination with tezacaftor, elexacaftor, and ivacaftor was approved (Trikafta). Structure and properties Tezacaftor (C26H27F3N2O6, Mr = 520.5 g/mol) exists as a white … Tezacaftor