Vertex backed for continued cystic fibrosis dominance with Trikafta replacement in good shape

Of the five late-stage pipeline products expected to be licensed in the seven major pharma markets for cystic fibrosis (CF) between 2020 and 2030, four will be CF transmembrane conductance regulator (CFTR) modulators, according to GlobalData.

Crucially, the data and analytics company predicts that US biotech firm Vertex Pharmaceuticals’ (Nasdaq: VRTX) next-generation triple combination, VX-121 + tezacaftor + VX-561 (deutivacaftor) is expected to replace Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor) as ‘gold standard’ treatment for CF.

Mandana Emamzadeh, healthcare analyst at GlobalData, said: “KOLs noted that superior efficacy and less-frequent dosing of Vertex’s VX-121 + tezacaftor + VX-561 will result in rapid uptake, with many patients shifting from Trikafta/Kaftrio, current standard of care. Furthermore, they supported development of more CFTR modulators for patients who develop intolerances or allergies - or indeed experience adverse events in response to established products.