The Pulmonary-Allergy Drugs Advisory Committee (Ad Comm) votes 13 - 2 in favor of a label expansion for Vertex Pharmaceuticals' cystic fibrosis med Kalydeco (ivacaftor) to include the R117H mutation. This expands the approved CF mutation list to ten. (PR source: http://t.co/514NrU3K9Q )
There is an estimated 1110 patients with the R117H mutation compared to 2700 with the G551D mutation which is the main mutation treated with Kalydeco. So this is a very significant development.
About Kalydeco (from: http://investors.vrtx.com/releasedetail.cfm?ReleaseID=875448 )
KALYDECO® (ivacaftor) is currently approved to treat more than 2,600 people ages 6 and older in North America, Europe and Australia who have specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In the United States, these mutations include G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. Cystic fibrosis is caused by a defective or missing CFTR protein that results from mutations in the CFTR gene. Multiple clinical studies are complete, underway or planned that are designed to evaluate whether ivacaftor, used alone or in combination with other potential CF medicines known as CFTR correctors, may help more people with CF, including people with one or two copies of the most common mutation, F508del. Data from several of these studies will be presented at NACFC, including the results of the Phase 3 TRAFFIC and TRANSPORT studies of the CFTR corrector lumacaftor in combination with ivacaftor, as well as the first interim data from a rollover study of patients who completed treatment in TRAFFIC and TRANSPORT. Other presentations include data from Phase 2 and 3 studies of ivacaftor and Phase 2 studies of VX-661, Vertex's second CFTR corrector, in combination with ivacaftor. Additionally, Vertex today announced that it plans to initiate a pivotal Phase 3 development program for VX-661 in combination with ivacaftor that will evaluate people with CF who have one or two copies of the F508del mutation, including people whose second CFTR mutation is known to cause a defect in the gating of the CFTR protein, pending regulatory discussions and data from an ongoing 12-week Phase 2b study of VX-661 in combination with ivacaftor in people with two copies of the F508del mutation.
"With KALYDECO, we have shown that treating the underlying cause of CF can have significant and sustained benefits for people with the G551D mutation," said Jeffrey Chodakewitz, M.D., Senior Vice President and Chief Medical Officer at Vertex. "Our goal is to develop combinations of medicines to treat many more people with CF and to improve the benefit that these combinations of medicines may provide. Data from the Phase 3 studies of lumacaftor in combination with ivacaftor showed consistent evidence of clinical benefit in lung function and other measures of the disease for people with two copies of the F508del mutation and provided important support to conduct further studies of combination regimens aimed at treating people with one and two copies of the F508del mutation."
- Chris
There is an estimated 1110 patients with the R117H mutation compared to 2700 with the G551D mutation which is the main mutation treated with Kalydeco. So this is a very significant development.
About Kalydeco (from: http://investors.vrtx.com/releasedetail.cfm?ReleaseID=875448 )
KALYDECO® (ivacaftor) is currently approved to treat more than 2,600 people ages 6 and older in North America, Europe and Australia who have specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In the United States, these mutations include G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. Cystic fibrosis is caused by a defective or missing CFTR protein that results from mutations in the CFTR gene. Multiple clinical studies are complete, underway or planned that are designed to evaluate whether ivacaftor, used alone or in combination with other potential CF medicines known as CFTR correctors, may help more people with CF, including people with one or two copies of the most common mutation, F508del. Data from several of these studies will be presented at NACFC, including the results of the Phase 3 TRAFFIC and TRANSPORT studies of the CFTR corrector lumacaftor in combination with ivacaftor, as well as the first interim data from a rollover study of patients who completed treatment in TRAFFIC and TRANSPORT. Other presentations include data from Phase 2 and 3 studies of ivacaftor and Phase 2 studies of VX-661, Vertex's second CFTR corrector, in combination with ivacaftor. Additionally, Vertex today announced that it plans to initiate a pivotal Phase 3 development program for VX-661 in combination with ivacaftor that will evaluate people with CF who have one or two copies of the F508del mutation, including people whose second CFTR mutation is known to cause a defect in the gating of the CFTR protein, pending regulatory discussions and data from an ongoing 12-week Phase 2b study of VX-661 in combination with ivacaftor in people with two copies of the F508del mutation.
"With KALYDECO, we have shown that treating the underlying cause of CF can have significant and sustained benefits for people with the G551D mutation," said Jeffrey Chodakewitz, M.D., Senior Vice President and Chief Medical Officer at Vertex. "Our goal is to develop combinations of medicines to treat many more people with CF and to improve the benefit that these combinations of medicines may provide. Data from the Phase 3 studies of lumacaftor in combination with ivacaftor showed consistent evidence of clinical benefit in lung function and other measures of the disease for people with two copies of the F508del mutation and provided important support to conduct further studies of combination regimens aimed at treating people with one and two copies of the F508del mutation."
- Chris