The cystic fibrosis (CF) market in the seven major markets (7MM) is forecast to grow from $11.1 billion in 2024 to $16.0 billion in 2034, growing at a modest compound annual growth rate (CAGR) of 3.8%, with therapies within the cystic fibrosis transmembrane conductance regulator (CFTR) modulator drug-class set to continue as a growth driver for the market over the forecast period, according to GlobalData, a leading data and analytics company.
GlobalData’s latest report, “Cystic Fibrosis Market Opportunity Assessment, Epidemiology, Clinical Trials, Unmet Needs and Forecast to 2034,” reveals that Vertex Pharmaceuticals’ dominance in the CF space is expected to continue over the forecast period, with the company’s CFTR modulator franchise expected to account for 85.5% of the overall market in 2034 across the 7MM.
Current treatment options for CF often require the use of therapies from multiple drug-classes, with CFTR modulators being the standard of care in the treatment paradigm. Vertex Pharmaceuticals’ CFTR modulator portfolio dominates the CF treatment landscape, with Trikafta/Kaftrio (elexacaftor + tezacaftor + ivacaftor and ivacaftor) currently the gold standard among the CFTR modulators.
Vinie Varkey, Healthcare Analyst at GlobalData, comments: “Vertex Pharmaceuticals’ CFTR modulator franchise takes a central place in CF treatment paradigm due to its first-to-market innovative therapy options that target the underlying defect causing CF and is available for a wide range of age groups (>1 month) and addresses a broad mutation coverage.
“Additionally, the company’s commitment to keep improving therapy options is evident through its strategy of progressively launching next-generation therapy options such as Alyftrek that needs to be administered once-daily, aiming to reduce treatment burden compared to earlier therapy options such as Trikafta that needs to be administered twice-daily. Alyftrek was approved in the US in December 2024, and in July 2025 in Europe and in Canada.”
According to GlobalData’s Drugs database, pipeline assets in development for CF draw attention to a limited range of mechanisms of action (MOAs), with over half of the clinical pipeline focusing on CFTR modulators. Examples include Sionna Therapeutics’ SION-719 (Phase I) and SION-451 (Phase I); the company is exploring nucleotide binding domain 1 (NBD1) approach to stabilize misfolded CFTR proteins during translation that aims to normalize the function of the CFTR protein, and this differs from Vertex’s post-translational correction strategy. Other new modalities that are being explored include mRNA therapy and gene therapy utilizing lentiviral and AAV-based vector platforms, representing potential treatment options that are mutation-agnostic.
Varkey adds: “Although emerging CFTR modulators in pipeline development represent a potential threat to Vertex, the overall dominance of this drug-class is expected to continue over the forecast period, thereby contributing the largest proportion to the growth of the CF market. Additionally other innovative, symptom management therapeutic approaches targeting alternative pathways are also being explored, including phage therapies to target lung infections and assets with mucolytic properties to improve mucus airway clearance, all of which highlight the use of multiple drug-classes for treatment of CF.”
While CFTR modulators are expected to lead the growth of the CF market, other factors such as anticipated increase in treatment rates resulting from rollout of newborn screening and genetic testing programs are also set to contribute to this growth. However, the high treatment costs associated with currently marketed CFTR modulators pose as a barrier to market access and wider adoption among patient population. Additionally, patent expiries of key therapies within the CFTR modulator drug-class are anticipated to lead to the entry of generic CFTR modulators over the forecast period.
Varkey concludes: “Looking ahead, the CF market is anticipated to benefit from emerging therapeutic modalities and precision medicine approaches, and the limited pipeline makes it an attractive indication with minimal competition from new or emerging players.”
*7MM – the US, France, Germany, Italy, Spain, the UK, and Canada
