A new treatment regimen for cystic fibrosis from Vertex Pharmaceuticals

“Vertex Pharmaceuticals” (Vertex Pharmaceuticals) has developed a new scheme for the treatment of cystic fibrosis – more effective and convenient to use than the combined drug “Trikafta” / “Caftrio”, which is now considered the most effective drug for the treatment of patients.

The new triple combination consists of drugs: vanzacaftor (vanzacaftor), tezacaftor (tezacaftor) and deutivacaftor (deutivacaftor). The therapeutic combination of these drugs is prescribed once a day, while “Trikafta” / “Caftrio” – in the morning and in the evening. The experimental treatment of cystic fibrosis has successfully passed clinical trials at the age of 6 years and older, and Vertex intends to register this gene modulator by mid-2024. The future name of the new combined drug announced by the manufacturer is Alyftrek.

A new triple therapy that replaces Trikafti/Caftrio promises additional improvements in patient health. In the future, Vertex Pharmaceuticals, with the support of Moderna, is working on inhaled VX-522 (Mrna-3692) experimental mRNA therapy for cystic fibrosis, which will deliver the genetic “instructions” encoding the synthesis of normal CFTR protein.

Treatment is aimed at those patients who have a complete lack of CFTR protein production, that is, CFTR gene modulators do not help them. This news gives hope to all patients with cystic fibrosis, regardless of mutations, for effective treatment and a full life.