Global Cystic Fibrosis (CF) Therapeutics 2020

                                          
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. Cystic fibrosis is a genetic disease that causes continuous lung infections and breathing problems over time.

Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, an ion channel involved in the transport of chloride and sodium ions across cell membranes.

While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s. People have 2 copies of this gene, one inherited from each parent and the disease only occurs when there is a mutation in both copies. CFTR is active in epithelial cells of organs such as of the lungs, pancreas, liver, digestive system, and reproductive tract. Alterations in the CFTR gene result in altered production, misfolding, or function of the protein and consequently abnormal fluid and ion transport across cell membranes. As a result, CF patients produce thick, sticky mucus that clogs the ducts of organs where it is produced making patients more susceptible to complications such as infections, lung damage, pancreatic insufficiency, and malnutrition.

The global Cystic Fibrosis (CF) Therapeutics market is valued at xx million US$ in 2020 is expected to reach xx million US$ by the end of 2029, growing at a CAGR of xx% during 2021-2029.

This report focuses on Cystic Fibrosis (CF) Therapeutics volume and value at global level, regional level and company level. From a global perspective, this report represents overall Cystic Fibrosis (CF) Therapeutics market size by analyzing historical data and future prospect. Regionally, this report focuses on several key regions: North America, Europe, China and Japan.

At company level, this report focuses on the production capacity, ex-factory price, revenue and market share for each manufacturer covered in this report.

The following manufacturers are covered:


 	
Vertex Pharmaceuticals
 	
Gilead
 	
AbbVie,
 	
Novartis AG
 	
F. Hoffmann-La Roche Ltd
 	
Alaxia
 	
Merck & Co.
 	
AIT (Advanced Inhalation Therapies)
 	
ALLERGAN
 	
AstraZeneca
 	
Teva Pharmaceutical Industries Ltd
 	
Alcresta

Segment by Type

 	
Pancreatic enzyme supplements
 	
Mucolytics
 	
Bronchodilators
 	
CFTR modulators

Segment by Route of treatment

 	
Oral drugs
 	
Inhaled drugs

Key Development in

In August 2020, Vertex Pharmaceuticals (Ireland) Limited received marketing authorization valid throughout the European Union for Kaftrio which is is a medicine used to treat patients aged 12 years and above who have cystic fibrosis, an inherited disease that has severe effects on the lungs, the digestive system and other organs. Kaftrio is an effective treatment for patients with cystic fibrosis who have two F508del mutations or one F508del and one MF mutation. Two of the active substances in Kaftrio, elexacaftor and tezacaftor, increase the number of CFTR proteins on the cell surface and the other, ivacaftor, improves the activity of the defective CFTR protein. These actions combine to make lung mucus and digestive juices less thick, thereby helping to relieve symptoms of the disease. Both are groups with a high unmet medical need. Patients with one F508del mutation plus other mutations were not covered in the studies, and although the company submitted some data on use in such patients, further data was considered necessary to support authorisation in these groups.

In terms of safety, Kaftrio was well tolerated. Therefore, the European Medicines Agency decided that Kaftrio’s benefits are greater than its risks and it can be authorised for use in the EU.