Cystic fibrosis market forecast to achieve $16.0bn by 2034 within the 7MM

The cystic fibrosis (CF) market throughout the seven main markets (7MM: the US, France, Germany, Italy, Spain, the UK, and Canada) is forecast to develop from $11.1bn in 2024 to $16.0bn in 2034, rising at a modest compound annual progress price of three.8%, as per GlobalData estimates. Whereas therapies from a number of drug courses are used for CF care, these throughout the cystic fibrosis transmembrane conductance regulator (CFTR) modulator drug class take central place within the remedy paradigm, with this mechanism of motion (MoA) additionally being probably the most distinguished one seen within the present pipeline panorama for CF and recognized as a continued progress driver for the CF market over the forecast interval.

Present remedy choices for CF typically require using therapies from a number of drug courses, with CFTR modulators as the usual of care within the remedy paradigm. Therapies throughout the CFTR modulator drug class goal particular mutations and affected person age teams, qualifying them as standard-of-care disease-modifying therapies inside CF. Vertex Prescribed drugs is the main participant in cystic fibrosis and its CFTR modulator portfolio dominates the CF remedy panorama, with Trikafta/Kaftrio presently the gold customary among the many CFTR modulators. Vertex’s most lately launched CFTR modulator, Alyftrek, is being positioned as a next-generation successor to Trikafta with a once-daily dosing routine that represents a lowered remedy burden in comparison with the latter’s twice-daily dosing routine. Vertex’s dominance within the CF area is predicted to proceed over the forecast interval, with the corporate’s CFTR modulator franchise anticipated to account for 85.5% of the general market in 2034 throughout the 7MM. Different supportive care remedy choices for CF embody pancreatic alternative therapies, mucolytics, inhaled antibiotics, and anti inflammatory therapies; use of those supplemental therapies is anticipated to cut back with uptake of CFTR modulators over the forecast interval.

Pipeline belongings in improvement for CF draw consideration to a restricted vary of MoAs. Over half of the scientific pipeline focuses on CFTR modulators. Rising CFTR modulators in pipeline improvement symbolize a possible risk to Vertex. An instance consists of Sionna Therapeutics’ SION-719 (Section I) and SION-451 (Section I); the corporate is exploring a nucleotide-binding area 1 method to stabilise misfolded CFTR proteins throughout translation that goals to normalise the perform of the CFTR protein, and this differs from Vertex’s post-translational correction technique. Different new modalities which are being explored embody messenger ribonucleic acid remedy and gene remedy utilising lentiviral and adeno-associated virus-based vector platforms, representing potential remedy choices which are mutation-agnostic. Examples embody Arcturus Therapeutics with its asset ARCT-032 (Section II), ReCode Therapeutics with its asset RCT-2100 (Section I/II), Vertex Prescribed drugs with its asset VX-522 (Section I/II), 4D Molecular Therapeutics with its asset 4D-710 (Section III (deliberate)), Spirovant Sciences with its asset SP-101 (Section I/II), and Boehringer Ingelheim with its asset BI 3720931 (Section I/II) that makes use of a lentiviral-based platform. Modern therapeutic approaches concentrating on different pathways are additionally being explored, together with phage therapies to focus on lung an infection and belongings with mucolytic properties to enhance mucus airway clearance.

Whereas CFTR modulators are anticipated to guide the expansion of the CF market, different components such because the anticipated enhance in remedy charges ensuing from the rollout of new child screening and genetic testing programmes are additionally set to contribute in the direction of this progress. Nonetheless, the excessive remedy prices related to presently marketed CFTR modulators pose a barrier to market entry and wider adoption amongst affected person populations. Moreover, patent expiries of key therapies throughout the CFTR modulator drug class are anticipated to result in the entry of generic CFTR modulators over the forecast interval. Wanting forward, the CF market is one that’s anticipated to profit from rising therapeutic modalities and precision drugs approaches, and the restricted pipeline makes it a pretty indication with minimal competitors from new/rising gamers.