Vertex announced positive interim data from the phase II data evaluating VX-809 and Kalydeco (ivacaftor) in cystic fibrosis patients with two copies of the F508del mutation.
The phase II trial is a randomized, double-blind, placebo-controlled study. The data released this time involved in 108 adult patients with cystic fibrosis with one or two copies of the F508del mutation. Patients were divided into five treatment groups of 20
patients each. Three groups of homozygous
One group of homozygous received VX-809 200 mg alone for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days
One group of homozygous received VX-809 400 mg alone for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days
One group of homozygous received VX-809 600 mg alone for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days
One group of heterozygous patients received VX-809 600 mg for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days.
One group of both homozygous and heterozygous patients received placebo.
The interim analysis included data from 37 homozygous F508del patients who completed treatment in the 56-day study and 11 patients with one or two copies of the F508del mutation who received placebo.
The results showed significant absolute improvement in lung function from baseline in patients treated with the combined treatments compared to patients who received placebo.
Significant reduction in sweat chloride was also seen in patients treated with VX-809 alone when compared to baseline at day 28.
Ivacaftor (Kalydeco) has been approved by the US FDA in 2012 for use in patients with Cystic Fibrosis ages 6 and older who have at least one cope of the G551D mutation in the CFTR genes. The European Medicines Agency is currently reviewing the Marketing Authorization Application submitted by Vertex in October of last year.
The phase II trial is a randomized, double-blind, placebo-controlled study. The data released this time involved in 108 adult patients with cystic fibrosis with one or two copies of the F508del mutation. Patients were divided into five treatment groups of 20
patients each. Three groups of homozygous
One group of homozygous received VX-809 200 mg alone for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days
One group of homozygous received VX-809 400 mg alone for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days
One group of homozygous received VX-809 600 mg alone for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days
One group of heterozygous patients received VX-809 600 mg for 28 days and then in combination with Kalydeco 250 mg for an additional 28 days.
One group of both homozygous and heterozygous patients received placebo.
The interim analysis included data from 37 homozygous F508del patients who completed treatment in the 56-day study and 11 patients with one or two copies of the F508del mutation who received placebo.
The results showed significant absolute improvement in lung function from baseline in patients treated with the combined treatments compared to patients who received placebo.
Significant reduction in sweat chloride was also seen in patients treated with VX-809 alone when compared to baseline at day 28.
Ivacaftor (Kalydeco) has been approved by the US FDA in 2012 for use in patients with Cystic Fibrosis ages 6 and older who have at least one cope of the G551D mutation in the CFTR genes. The European Medicines Agency is currently reviewing the Marketing Authorization Application submitted by Vertex in October of last year.
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