Vertex’s triple combination CFTR modulators drive cystic fibrosis market to $12.7bn by 2030, says GlobalData

The cystic fibrosis (CF) market is expected to grow from $8.2bn in 2020 to $12.7bn in 2030 in the seven major markets (7MM*) at a compound annual growth rate (CAGR) of 4.5%, according to GlobalData. The leading data and analytics company notes that significant growth in this market will be driven by the uptake of Vertex Pharmaceutical’s CF transmembrane conductance regulator (CFTR) modulators, Trikafta/Kaftrio and VX-121 + tezacaftor + VX-561 (deutivacaftor), both high-priced triple combination therapies.

The company’s latest report, ‘Cystic Fibrosis: Global Drug Forecast and Market Analysis to 2030’, reveals that another key factor driving an increase in the CF market is the continued growth of the patient population, as well as the improving life expectancy of CF patients.

Mandana Emamzadeh, PhD, Healthcare Analyst at GlobalData, comments: “CFTR modulators are preferred over other CF drugs, because they target the underlying cause of the disease. Vertex is the first and only company to bring these types of therapies to market and has revolutionized the space yet again with the recent launch of Trikafta/Kaftrio. With its unparalleled efficacy across a wide range of CF patients, Trikafta/Katrio is quickly becoming the gold standard.”

GlobalData expects that increasing uptake of Trikafta/Kaftrio over the forecast period will cannibalize sales from Vertex’s other three CFTR modulator products, with sales of Kalydeco, Orkambi, and Symdeko/Symkevi shrinking from $2.2bn in 2020 to $46.9m by 2030. Global sales for Trikafta/Kaftrio are projected to peak at $7.2bn in 2024 before the launch of Vertex’s next-generation triple-combination therapy, VX-121 + tezacaftor + VX-561 (deutivacaftor), in 2025. Once launched, this drug is expected to show strong uptake and achieve global sales of $8.8bn in 2030, representing nearly 70% of the total market size.

Emamzadeh continues: “Based on insight from key opinion leaders (KOLs) interviewed by GlobalData, increasing uptake of these triple-combination CFTR modulator therapies is expected to greatly improve the overall lung health of CF patients and consequently decrease the use of many symptomatic therapies including inhaled antibiotics and mucolytics. Total sales for inhaled antibiotics and mucolytic drugs will drop from 17.9% ($1.1bn) and 7.3% ($465.5m) of total sales in 2020 to 10.7% ($917.4m) and 4.9% ($424.1m) of total sales in 2030, respectively. In contrast, the prescribing rate of pancreatic enzyme replacement therapy is not expected to be greatly impacted by triple combinations during the forecast period.”

GlobalData’s report highlights five products within the late-stage pipeline for CF: four CFTR modulators, two by Vertex, VX-561 (deutivacaftor) and VX-121 + tezacaftor + VX-561, and two by AbbVie, ABBV-3067 and ABBV-3067 + galicaftor (ABBV-2222), as well as an anti-inflammatory drug, LAU-7b (fenretinide) by Laurent Pharmaceuticals.

Emamzadeh explains: “Vertex’s VX-121 + tezacaftor + VX-561 demonstrated favorable tolerability and strong efficacy compared to Trikafta/Kaftrio in a late-stage Phase II trial. KOLs believed that this therapy could actually top the stellar clinical profile of Trikafta/Kaftiro.

“In contrast, KOLs did not expect that AbbVie’s dual combination product, ABBV-3067 + galicaftor, would be superior to Trikafta/Kaftrio and believed that AbbVie’s pipeline products would more likely be positioned as alternatives for patients who cannot tolerate established CFTR modulator therapies. Finally, due to a lack of publicly available data, uncertainty remains regarding the overall clinical profile of Laurent’s LAU-7b (fenretinide), the only anti-inflammatory pipeline agent in late-stage clinical development.”

Anticipated barriers to growth in the CF market between 2020 and 2030 include slow reimbursement for novel CFTR modulators in some 5EU countries and Canada, lack of novel products in the mucolytic or inhaled antibiotic class, and decreased usage of symptomatic drug classes following widening adoption of triple combination therapies.

Emamzadeh concludes: “Although important, these barriers will have a limited impact on growth. The market is expanding at an impressive rate and ample opportunities exist for developers to further improve the CF treatment landscape.”

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

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