New Class of Drugs
Cystic Fibrosis Market to Triple by 2025
By Ilene Schneider
Thanks to new drugs, the cystic fibrosis (CF) market in the US, France, Germany, Italy, Spain, the UK and Canadawill more than triple from $2.1 billion in 2015 to $7.6 billion by 2025, with a compound annual growth rate of 13.6percent, according to research and consulting firm GlobalData.
Global Data’s latest report,OpportunityAnalyzer: Cystic Fibrosis – Opportunity Analysis and Forecasts to 2025, predicts that 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, will drive the growth.Insmed's inhaled antibiotic Arikace (liposomal amikacin) is not expected to drive CF market growth as much as the CFTR modulator, because it will compete with other inhaled antibiotics.
CF is an autosomal recessive disease caused by mutations in the CFTR gene, which codes for the CFTR protein. Patients present a variety of clinical symptoms. The disease primarily affects the respiratory and gastrointestinal tracts.
Historically, three classes of drugs have been used to for treatment in CF patients: inhaled antibiotics for bacterial and fungal respiratory infections, mucolytic drugs for mucociliary clearance of the airways and pancreatic enzyme replacement therapies (PERTs) for treatment of CF induced exocrine pancreatic insufficiency (EPI). During the last five years, a new class of drugs specifically treating the underlying cause of CF was launched into the market, helping to restore function of the insufficient CFTR protein.
According to PavanKottamasu, MSc, healthcare analyst for GlobalData, “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.
Kottamasu added, “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.”
In spite of major improvement in the treatment of cystic fibrosis, patients are expected to encounter challenges 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. Still, according to Kottamasu,“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.”