Vertex Receives CHMP Positive Opinion for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), a new once-daily CFTR Modulator for the treatment of cystic fibrosis

Vertex Pharmaceuticals announced that the  EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

“Our goal has always been to serially innovate to help people with cystic fibrosis live healthier and longer lives. If approved, this new medicine would be indicated for people with CF ages 6 years and older with at least one non-class I mutation, meaning more patients would be eligible for a medicine that gets them closer to normal levels of sweat chloride,” said Dr. Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs,and Chief Medical Officer, Vertex.

Alyftrek will be available as 50 mg / 20 mg / 4 mg and 125 mg / 50 mg / 10 mg film-coated tablets. The active substances of Alyftrek are deutivacaftor / tezacaftor / vanzacaftor (ATC code: R07AX33). Vanzacaftor and tezacaftor, are CFTR correctors that bind to different sites on the CFTR protein, leading to an increase in the amount of CFTR protein on the cell surface; deutivacaftor improves the activity of the defective CFTR protein at the cell surface. These combined actions make lung mucus and digestive juices less thick, thereby helping to relieve symptoms of the disease.

The benefits of Alyftrek are improved lung function, as measured by percent predicted FEV1  (forced expiratory volume in one second), after 24 weeks of treatment. Two phase III, randomised, double-blind clinical studies showed Alyftrek to be as effective as Kaftrio in patients with cystic fibrosis and non-class I mutations aged 12 years and older. A supportive, single arm, open-label study in children aged 6-11 years was also submitted. Most children harboured an F508del mutation.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official EU languages after the marketing authorisation has been granted by the European Commission.

"CFTR modulators have already revolutionized the way we treat CF and I am encouraged that, if approved, this medicine could advance CF treatment even further,” said Prof. Marcus A.  Chairman of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité - Universitätsmedizin Berlin. “The results we saw from the two deutivacaftor/tezacaftor/vanzacaftor Phase 3 clinical trials were motivating as they showed non-inferiority in ppFEV₁ and superior improvement of sweat chloride levels compared to ivacaftor/tezacaftor/elexacaftor in combination with ivacafto