The cystic fibrosis (CF) market across the seven major markets (7MM: the US, France, Germany, Italy, Spain, the UK, and Canada) is forecast to grow from $11.1bn in 2024 to $16.0bn in 2034, growing at a modest compound annual growth rate of 3.8%, as per GlobalData estimates. While therapies from multiple drug classes are used for CF care, those within the cystic fibrosis transmembrane conductance regulator (CFTR) modulator drug class take central place in the treatment paradigm, with this mechanism of action (MoA) also being the most prominent one seen in the current pipeline landscape for CF and identified as a continued growth driver for the CF market over the forecast period.
Current treatment options for CF often require the use of therapies from multiple drug classes, with CFTR modulators as the standard of care in the treatment paradigm. Therapies within the CFTR modulator drug class target specific mutations and patient age groups, qualifying them as standard-of-care disease-modifying treatments within CF. Vertex Pharmaceuticals is the leading player in cystic fibrosis and its CFTR modulator portfolio dominates the CF treatment landscape, with Trikafta/Kaftrio currently the gold standard among the CFTR modulators. Vertex’s most recently launched CFTR modulator, Alyftrek, is being positioned as a next-generation successor to Trikafta with a once-daily dosing regimen that represents a reduced treatment burden compared to the latter’s twice-daily dosing regimen. Vertex’s 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. Other supportive care treatment options for CF include pancreatic replacement therapies, mucolytics, inhaled antibiotics, and anti-inflammatory therapies; use of these supplemental therapies is anticipated to reduce with uptake of CFTR modulators over the forecast period.
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Pipeline assets in development for CF draw attention to a limited range of MoAs. Over half of the clinical pipeline focuses on CFTR modulators. Emerging CFTR modulators in pipeline development represent a potential threat to Vertex. An example includes Sionna Therapeutics’ SION-719 (Phase I) and SION-451 (Phase I); the company is exploring a nucleotide-binding domain 1 approach to stabilise misfolded CFTR proteins during translation that aims to normalise the function of the CFTR protein, and this differs from Vertex’s post-translational correction strategy. Other new modalities that are being explored include messenger ribonucleic acid therapy and gene therapy utilising lentiviral and adeno-associated virus-based vector platforms, representing potential treatment options that are mutation-agnostic. Examples include Arcturus Therapeutics with its asset ARCT-032 (Phase II), ReCode Therapeutics with its asset RCT-2100 (Phase I/II), Vertex Pharmaceuticals with its asset VX-522 (Phase I/II), 4D Molecular Therapeutics with its asset 4D-710 (Phase III (planned)), Spirovant Sciences with its asset SP-101 (Phase I/II), and Boehringer Ingelheim with its asset BI 3720931 (Phase I/II) that uses a lentiviral-based platform. Innovative therapeutic approaches targeting alternative pathways are also being explored, including phage therapies to target lung infection and assets with mucolytic properties to improve mucus airway clearance.
While CFTR modulators are expected to lead the growth of the CF market, other factors such as the anticipated increase in treatment rates resulting from the rollout of newborn screening and genetic testing programmes are also set to contribute towards this growth. However, the high treatment costs associated with currently marketed CFTR modulators pose a barrier to market access and wider adoption among patient populations. 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. Looking ahead, the CF market is one that 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/emerging players.
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