High prices for combination regimens will translate to high revenues, despite the introduction of generics in the mid-term future. The increased use of Trikafta will offset the decrease in revenues caused by generics over the forecast period. 

The first CFTR modulator triplet, Trikafta, is approved in patients with at least one F508del mutation regardless of their second mutation. Despite its late approval, Trikafta is the first CFTR modulator approved for F508del heterozygous patients, and its substantial improvement in lung function over both Symdeko and Orkambi in F508del homozygous patients has allowed it to capture the majority of the CF patient population. 

In December 2024, Alyftrek’s FDA approval positions it as a likely successor to Trikafta, particularly because it offers the advantage of once-daily dosing and leverages Vertex’s expertise in the CFTR modulator market. Clinical trial results revealed that Alyftrek is comparable to Trikafta in improving lung function and surpasses Trikafta in reducing sweat chloride levels.

This Datamonitor Healthcare report contains a Disease Analysis module.