The cystic fibrosis (CF) space across the seven major markets––the United States, France, Germany, Italy, Spain, the United Kingdom, and Canada––is set to more than triple from $2.1 billion in 2015 to approximately $7.6 billion by 2025, representing a compound annual growth rate of 13.6%, according to an analysis from consulting firm GlobalData.
The company’s latest report states that the major drivers of this substantial growth included the introduction of Orkambi (ivacaftor/lumacaftor, Vertex Pharmaceuticals) in 2015, followed by the introduction of new, high-priced disease-modifying cystic fibrosis transmembrane conductance regulator (CFTR) modulators during the forecast period.
The inhaled antibiotic Arikace (liposomal amikacin, Insmed) is also expected to launch during the forecast period, but it is not expected to drive CF market growth as much as the CFTR modulators because it will be competing with other inhaled antibiotics.
CF is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which codes for the CFTR protein. The disease mainly affects the respiratory and gastrointestinal tracts.
Three classes of drug are currently used to treat patients with CF: inhaled antibiotics for bacterial and fungal respiratory infections; mucolytic drugs for mucociliary clearance of the airways; and pancreatic enzyme replacement therapies (PERTs) for the treatment of CF-induced exocrine pancreatic insufficiency (EPI). During the last five years, CFTR modulators have improved the treatment of CF patients by helping to restore the function of insufficient CFTR proteins.
Analyst Pavan Kottamasu explains: “Until recently, R&D strategies in the cystic fibrosis space have targeted organ-specific sequelae of the underlying disease. This approach has mainly resulted in the development of new formulations of antibiotics that can be administered via inhalation for the management of chest infections, and therapies that enhance airway mucociliary clearance.
“However, over the last decade, advanced technologies have yielded a shift in research development towards therapies that target the underlying cause of the disease by enhancing CFTR function. Currently, Vertex controls the entire CFTR-modulator market share with its two marketed CFTR modulators, Kalydeco (ivacaftor) and Orkambi.
“There is also a trend towards licensing products, and alliances are being pursued by a wide spectrum of pharmaceutical and biotechnology manufacturers, as the majority of novel therapeutic agents for cystic fibrosis are being developed by small companies. GlobalData anticipates that licensing activity and the formation of new partnerships will steer smaller companies to continue researching novel compounds.”
Despite recent advances, the overall life expectancy of CF patients is still 20–25 years less than that of the healthy population. Moreover, patients still face considerable unmet needs because of the lack of curative therapies; the limited choice of mucolytic therapies; and the constant fight against lung infection that requires the development of novel classes of antibiotics, the report says.
Kottamasu continues: “Following the approval of Kalydeco and Orkambi, there is currently enormous excitement for CFTR modulators that can modify the course of the disease and potentially turn this life-threatening condition into a chronic one. At the same time, opportunity remains for newer drugs to improve upon the efficacy of the current marketed and late-stage pipeline CFTR modulators.”
Source: GlobalData; April 4, 2017.