The cystic fibrosis space across the seven major markets of the US, France, Germany, Italy, Spain, the UK, and Canada, is set to more than triple from $2.1 billion in 2015 to around $7.6 billion by 2025, representing a compound annual growth rate of 13.6%, according to research and consulting firm GlobalData.
The company’s latest report states that a major driver of this substantial growth includes the introduction of Vertex’s Orkambi (ivacaftor + lumacaftor) in 2015, followed by the introduction of novel, high-priced, disease-modifying cystic fibrosis transmembrane conductance regulator (CFTR) modulators during the forecast period.
Pavan Kottamasu, MSc, Healthcare Analyst for GlobalData, 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 significant improvement in the treatment of cystic fibrosis, patients will still face considerable unmet needs due to 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.
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.”