Global Cystic Fibrosis Therapeutics Market Size, Share, Growth Analysis, By Drug Class(CFTR modulators, Mucolytics), By Route of Administration(Oral drugs and Inhaled drugs) - Industry Forecast 2024-2031

In May 2023, the Cystic Fibrosis Foundation announced the success of their $1 billion fund raise with the purpose of new treatment research for cystic fibrosis. The funding will support research into gene editing, gene therapy, and other promising new technologies.

In April 2023, Vertex Pharmaceuticals announced that it has entered into a collaboration with CRISPR Therapeutics to develop gene editing therapies for cystic fibrosis. The collaboration will focus on developing CRISPR-based therapies that target the underlying genetic defects that cause cystic fibrosis.

In March 2023, AbbVie announced that the FDA granted fast track designation to its investigational drug ABBV-555 for the treatment of cystic fibrosis. ABBV-555 is a small molecule corrector that is designed to target the G551D mutation, the second most common mutation that causes cystic fibrosis.

In February 2023, Shionogi announced that the FDA granted orphan drug designation to its investigational drug SHP616 for the treatment of cystic fibrosis. SHP616 is a small molecule corrector that is designed to target the F508del mutation, the most common mutation that causes cystic fibrosis.

In January 2023, Vertex Pharmaceuticals announced that the FDA approved Trikafta (elexacaftor/ivacaftor/tezacaftor), a triple combination therapy for the treatment of cystic fibrosis in patients 12 years of age and older. Trikafta is the first and only triple combination therapy for cystic fibrosis and has been shown to significantly improve lung function and quality of life in patients.

In October 2021, Vertex Pharmaceuticals announced that the European Medicines Agency (EMA) had accepted the Marketing Authorization Application (MAA) for their investigational triple combination therapy for cystic fibrosis, VX-121/tezacaftor/ivacaftor.

In August 2021, Vertex Pharmaceuticals received approval from the U.S. Food and Drug Administration (FDA) for their triple combination therapy, Trikafta, for patients with cystic fibrosis ages 6 through 11 who have at least one F508del mutation.