Vertex Reports Positive Phase 3 Results for Trikafta in People 12 and Older with Certain CF Mutations
July 21, 2020
Vertex Pharmaceuticals reported positive results of a global phase 3 study of its triple combination therapy Trikafta in people with cystic fibrosis ages 12 years and older as part of its post-marketing commitment to the U.S. Food and Drug Administration.
The study was in patients who have one copy of the F508del mutation and one gating mutation (F/G) or one copy of the F508del mutation and one residual function mutation (F/RF).
The study met its primary endpoint and all secondary endpoints. The regimen was generally well-tolerated, and safety data were consistent with those observed in previous phase 3 studies with Trikafta.
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. While there are many different types of CFTR mutations that can cause the disease, most people with CF have at least one F508del mutation. These mutations lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.
Trikafta, a combination of elexacaftor/tezacaftor/ivacaftor, is used for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Trikafta is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The approval of Trikafta was supported by positive results of two global phase 3 studies in people ages 12 years and older with CF.
The newly reported phase 3 study is a post-marketing
commitment in the United States and the results will be submitted to the U.S.
Food and Drug Administration.
In June, Vertex received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for the initial triple combination regimen application for people with CF ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F). Data from Vertex’s latest study will be submitted to the European Medicines Agency to support a potential indication expansion of the EU label, once European Commission approval has been granted for the initial triple combination application.
“The results of this study demonstrate that the triple
combination provides significant additional benefit compared to existing CFTR
modulator therapy for F/G and F/RF patients and adds to the robust body of
evidence supporting the benefit of this medicine for patients with at least one
F508del mutation,” said Carmen Bozic, executive vice president of global
medicines development and medical affairs and chief medical officer at Vertex.
Carmen Bozic, executive vice president of global medicines development and medical affairs and chief medical officer at Vertex
Author: Rare Daily Staff
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