Vertex and AbbVie are the only current and future players in the cystic fibrosis market, says GlobalData

The disease-modifying nature of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has transformed the treatment paradigm for cystic fibrosis (CF) over the last decade, a trend that is likely to persist to 2030, and will greatly improve the quality of life and longevity of patients, according to GlobalData. The leading data and analytics company notes that Vertex is the clear market leader, dominating the CF space with four marketed CFTR modulators.

Marketed drugs currently available for 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 CFTR modulators enhance CFTR function, targeting the underlying cause of disease.

Mandana Emamzadeh, PhD, Pharma Analyst at GlobalData, comments: “There are over a dozen pharmaceutical companies that are current players in the CF market, but Vertex and AbbVie are the only current and future players. Vertex currently dominates the CF space with four marketed CFTR modulators available throughout the seven major market (7MM*), including Kalydeco, Orkambi, Symdeko/Symkevi, and Trikafta/Kaftrio.

“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 may currently own the CFTR modulator drug class, other players stand out with marketed products in other drug classes. Chiesi possesses two inhaled antibiotic products, Bramitob/Bethkis (US and 5EU**) and Quinsair (5EU); a mucolytic, Bronchitol (US); and a PERT product, Pertzye (US). Two other key companies include Viatris (formerly Mylan), which owns the most widely used inhaled antibiotic therapies, TOBI and TOBI Podhaler, and Nestlé Health Science, which owns two PERT products, Zenpep and Viokace. 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.

Emamzadeh adds: “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 to less than 12 months in Canada, and Trikafta/Kaftrio to pediatric CF patients between the ages of two and five in the US, and two and 11 years in the 5EU and Canada. Additionally, two new products from this company are expected to launch within next five years: VX-561 and VX-121 + tezacaftor + VX-561. At present, AbbVie markets Creon, the most widely used PERT by CF patients. The company has two pipeline agents, ABBV-3067, a CFTR potentiator, which 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 presently developing an anti-inflammatory agent, LAU-7b (fenretinide), for reducing the levels of inflammation and the severity of pulmonary infections with P. 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.

*7MM = US, France, Germany, Italy, Spain, UK, and Canada

**5EU = France, Germany, Italy, Spain, and UK

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