The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ARCT-032, Arcturus Therapeutics’ inhaled experimental treatment for cystic fibrosis (CF). The designation is intended to encourage the development of therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides incentives,…

The European Commission has expanded the approval of Kaftrio (ivacaftor, tezacaftor, and elexacaftor) in combination with Kalydeco (ivacaftor) to include children ages 2 to 5 with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene. Previously cleared to treat patients ages 6 and older…

Cystic fibrosis (CF) patients who are positive for the fungus Aspergillus and develop an allergic reaction to it experience worse outcomes after getting COVID-19, a study reports. The allergic response, called bronchopulmonary aspergillosis (ABPA), increases the risk of hospitalization and sepsis, a life-threatening immune system reaction to an infection, data show.

Kaftrio (elexacaftor/tezacaftor/ivacaftor) — known as Trikafta in the U.S. — brings broad benefits in the form of better lung function, quality of life, and cognitive processing, while also helping to ease depression and improve stool frequency for cystic fibrosis (CF) patients, a real-world study in Italy found. For…