Sales of CFTR modulators now constitute two thirds of the cystic fibrosis market.
By Daniel Chancellor, Lead Analyst
1 October 2016
I am a Lead Analyst covering the CNS and rare disease markets at Datamonitor Healthcare. I joined in early 2010, having ...
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Datamonitor Healthcare projects total sales of $2.6bn in 2016 in the US and five major EU markets, two thirds of which originate from Vertex’s portfolio of CFTR modulators. Kalydeco and Orkambi are forecast to generate $1.8bn, despite Kalydeco being first available in 2012 and Orkambi only available since 2015. This new class of drugs has revolutionized the treatment of cystic fibrosis, bringing specific treatments to patients tailored to individual genotypes, and has also transformed the market. The dominance of the CFTR modulator class lies in the price premium that Vertex is able to charge – these products have no comparators against which to benchmark, but instead produce lung function improvements in addition to existing antibiotic and mucolytic treatments. The wholesale acquisition price of Kalydeco in the US is $312,000 per year, while Orkambi costs $260,000 per year; both greatly exceed the prices of comparators in other classes such as Pulmozyme (dornase alfa; Roche) and Tobi Podhaler (tobramycin dry powder inhaler; Novartis), which have annual costs of approximately $40,000 and $50,000, respectively (Red Book, 2016).
The CFTR modulator class is expected to continue to expand throughout the forecast period, as current treatments become available to new patients and the next generation of CFTR-targeting drugs emerges from the pipeline. By 2025, Datamonitor Healthcare expects the class to be worth $5.5bn. Initial growth will be relatively tempered as Vertex has already maximized its addressable population for Kalydeco, although improved market access for Orkambi in Europe, together with an expansion of its label to include patients in the 6–11 years and 2–5 years age groups, will allow for increased adoption. The launch of subsequent CFTR modulators in 2018, including Vertex’s own VX-661/ivacaftor and PTC Therapeutics’ drug for nonsense mutation cystic fibrosis Translarna (ataluren), will allow for a greater range of cystic fibrosis genotypes to have specific treatment options. These two new drugs, especially VX-661/ivacaftor, will greatly propel the CFTR modulator class value.
Datamonitor Healthcare’s Cystic Fibrosis: Forecast module uses a patient-based forecasting approach to estimate the sales of key marketed and late-phase pipeline drugs across the US and five major EU markets (France, Germany, Italy, Spain, and the UK) during 2016–25.
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