The currently available marketed drugs for cystic fibrosis (CF) target multiple aspects of the disease. Inhaled antibiotics are used for the treatment of chronic lung infections, mucolytic agents are used for reducing the viscosity of the mucus in the lungs, pancreatic enzyme replacement therapies (PERTs) are used for the treatment of CF-related exocrine pancreatic insufficiency (EPI), and CF transmembrane conductance regulator (CFTR) modulators enhance CFTR function, targeting the underlying cause of disease. The disease-modifying nature of CFTR modulators has transformed the treatment paradigm for CF over the last decade, setting a trend that is likely to persist to 2030.
More than a dozen pharmaceutical companies make up the current players in the CF market (Figure). Vertex is the clear market leader, dominating the CF space with four marketed CFTR modulators available throughout the seven major markets (7MM) (US, France, Germany, Italy, Spain, UK and Canada), including Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor), Symdeko/Symkevi (tezacaftor/ivacaftor and ivacaftor), and Trikafta/Kaftrio (elexacaftor, tezacaftor, ivacaftor). Since the global approval of Kalydeco in 2012, Vertex has greatly expanded the number of CF-related mutations that are eligible for CFTR modulator treatment across all age groups. Currently, Vertex’s Trikafta/Kaftrio is the world’s first triple-combination therapy for which approximately 90% of the CF population will likely be eligible.
While Vertex is currently the only company with products in the CFTR modulator drug class, other players stand out with marketed products in other drug classes. Chiesi possesses two inhaled antibiotic products, Bramitob/Bethkis (tobramycin) in the US and five major European markets (5EU) (France, Germany, Italy, Spain and the UK) and Quinsair (levofloxacin) in the 5EU, as well as the mucolytic Bronchitol (mannitol) in the US and the PERT product Pertzye (pancrelipase) in the US. Two other key companies include Viatris (formerly Mylan), which owns the most widely-used inhaled antibiotic therapies, TOBI (tobramycin) and TOBI Podhaler (tobramycin inhalation powder), and Nestlé HealthScience, which owns two PERT products, Zenpep (pancrelipase) and Viokace (pancrelipase). The rest of the pharmaceutical companies with marketed drugs used for the treatment of CF include Gilead, Teva, Genentech, AbbVie, Vivus, Horizon Therapeutics and Pharmaxis.
Vertex and AbbVie are the only two companies with clear stakes in both the current and future CF market. Vertex is currently pursuing label expansion for Kalydeco to CF patients ages 0 months to less than 12 months in Canada, and Trikafta/Kaftrio to pediatric CF patients ages 2-5 years in the US and 2-11 years in the 5EU and Canada. Additionally, two new products from this company are expected to launch within the next five years: VX-561 and VX-121 + tezacaftor + VX-561. Currently, AbbVie markets Creon, the most widely used PERT by CF patients. The company has two pipeline agents, ABBV-3067, a CFTR potentiator that is being studied in late-stage Phase II trials alone and in combination with galicaftor (ABBV-2222), a CFTR corrector. If successful, AbbVie will be Vertex’s first competitor in the CFTR modulator market.
Laurent Pharmaceuticals is expected to be the only new player in the CF space by 2030. It is currently developing an anti-inflammatory agent, LAU-7b (fenretinide), for reducing the levels of inflammation and the severity of pulmonary infections with Pseudomonas aeruginosa. While this pipeline agent has an innovative mechanism of action and is a first-in-class drug, the company is a novice within the CF field and has not yet brought a drug to market.
Due to the limited number of pipeline agents in late-stage development, GlobalData anticipates that the current players in the CF market will likely maintain their competitive positions by 2030.