Vertex’s triple therapy will reshape the landscape of cystic fibrosis trials

Trikafta approval extends Vertex’s dominance, pushes competitors to rare mutations and new trial designs

FDA approval of Vertex’s long-awaited triple therapy for cystic fibrosis is likely to seal the company’s dominance in the indication for years to come, with competitive threats barely touching the horizon. Moreover, uptake of the triple therapy will make designing and enrolling future trials much harder.

The best plays left for competitors are to focus on the 10% of patients not treated by approved drugs, all of which Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) has brought to the scene, or to upstage the small molecules with new modalities like mRNA or one-and-done curative approaches.

The Cystic Fibrosis Foundation is setting its sights on the former. On Oct. 30 it announced “Path to a Cure” -- a $500 million commitment through 2025, to fund R&D therapies for patients whose genotypes aren’t treated by Vertex’s marketed drugs. (see “Cystic Fibrosis Foundation to Invest $500M”).

Until now, Vertex’s therapies addressed only half of the CF population, and while Orkambi lumacaftor/ivacaftor was a breakthrough when approved, some doctors and patients have not been impressed by its efficacy.

The launch of Trikafta elexacaftor/tezacaftor/ivacaftor sets a new gold standard for disease-modifying therapies for CF and solidifies Vertex’s leadership position in the market.

Trikafta’s efficacy data have been far superior to any other clinical readouts, including those of Vertex’s doublet therapies Orkambi and Symdeko tezacaftor/ivacaftor (see Table: “Cystic Fibrosis Clinical Data Comps”).


Figure: Cystic Fibrosis Clinical Data Comps

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