NEW YORK – The US Food and Drug Administration has accepted Vertex Pharmaceuticals' new drug application for a triplet CFTR modulator regimen for cystic fibrosis caused by certain genetic variants, the company said Tuesday.
The FDA granted priority review to the application and expects to issue a decision by Jan. 2, 2025, on the investigational cystic fibrosis therapy, a daily regimen comprising vanzacaftor, tezacaftor, and deutivacaftor.
Boston-based Vertex is seeking regulatory approval for the combination, dubbed "vanza triple," in cystic fibrosis patients who are at least 6 years old and who have certain variants in the CFTR gene, including F508del mutations. Separately, the European Medicines Agency has also validated Vertex's marketing authorization application for the combination in the same indication.
In cystic fibrosis, CFTR mutations lead to a dysfunctional CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface, while deutivacaftor aims to improve the flow of salt and water across the cell membrane by increasing the probability of the CFTR protein channel remaining open.
Tezacaftor is also part of another Vertex cystic fibrosis oral regimen, Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), approved in the US. It's also approved in the EU, where the regimen is branded as Kaftrio (elexacaftor/tezacaftor/ivacaftor) in combination with Kalydeco (ivacaftor).
"Vanzacaftor raises the high bar set by Trikafta and gives more people with CF the chance to get to levels of sweat chloride below the diagnostic threshold for CF, and even to levels of sweat chloride seen in those without CF," Nia Tatsis, Vertex's executive VP and chief regulatory and quality officer, said in a statement.
Vertex has also submitted regulatory applications for vanza triple in Canada, Australia, Switzerland, and the UK.