FDA accepts new drug applications for VX-445 (elexacaftor), tezacaftor and ivacaftor combination therapies

recently, Vertex Pharmaceuticals(http:// announcedthat the U.SFDA(http://accept thenewdrug(http:// http://application (NDA) for the combination of vX-445, tezacaftandandandandandandandandandandandandandandandapato
r, a combination ofand http:// the combination of the drugand the composition of the therapy, for the treatment of cystic fibrosis (NDA)The FDA's grant of this NDA priority review is expected to be in response by March 19, 2020Thisabout VX-445, tezacaftor and ivacaftor
Vertex developed VX-445 is a new generation of CFTR protein rectors that restore the function of CFTR proteins that carry F508del mutations, thereby improving the respiratory function of CF patients 　　Tezacaftor can enhance CFTR protein function by increasing the level of CFTR protein transport to the cell surface, while ivacaftor can improve the function of the defective CFTR protein by extending the opening hours of the CFTR protein on the cell surface The triple therapy was approved by the FDA in May 2018 　　The application was supported by two positive results from the World Phase 3 Clinical Trial (http:// for patients aged 12 and over 　　In the first 24-week randomized, double-blind, placebo-controlled Phase 3 clinical trial, VX-445 was triplethed with tezacaftor and ivacaftor to treat patients who carried a Mutation of the F508del gene and a functional lysage-minimizing gene mutation 　　The results showed that after 4 weeks of treatment, the patients in the treatment group had significantly improved lung function compared to the control group who received placebo treatment, showing an improvement of 13.8% (p0.001 ) in the average absolute value of the percentage of the expected amount of one-second force exhalation volume (ppFEV1) compared to the baseline In a four-week Phase 3 clinical trial, CF patients with two copies of the F508del CFTR gene were treated with VX-445 and tezacaftor and ivacaftor, or a placebo-triple therapy with tezacaftor and ivator.