Phase 3 Results from Two Studies of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Triple Combination Treatment for Cystic Fibrosis Concurrently Published in The New England Journa
- Both studies met primary and all key secondary endpoints demonstrating significant improvements in lung function and other measures of the disease -
- Results of both studies to be presented today at the 33rd Annual?North American Cystic Fibrosis Conference?as part of six presentations from Vertex at the meeting -
BOSTON--(BUSINESS WIRE)--Oct. 31, 2019--?Vertex Pharmaceuticals Incorporated?(Nasdaq: VRTX) today announced the concurrent publication in?The?New England Journal of Medicine?(NEJM)and?The Lancet?of results from two Phase 3 studies of TRIKAFTA? (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one?F508del?mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation. Data highlighting primary and secondary endpoints from a 24-week Phase 3 study in 403 people with one?F508del?mutation and one minimal function mutation (F/MF) in the?CFTR?gene were published in?NEJM. In addition,?The Lancetpublished data highlighting primary and secondary endpoints from a 4-week Phase 3 study in 107 people with two?F508del?mutations (F/F). Both studies met primary and all key secondary endpoints, demonstrating statistically significant and clinically meaningful improvements in lung function and other measures of disease. In these studies, TRIKAFTA was generally well tolerated. ?The results of the TRIKAFTA studies published in both?The Lancet?and?NEJM?are impressive and represent a historic moment in CF care, with the medicine demonstrating improvements in multiple CF outcome measures in clinical trials, while being generally well tolerated,? said?Raksha Jain, M.D., M.S.C.I., Associate Professor, Internal Medicine, Pulmonary and Critical Care, The?University of Texas Southwestern Medical Center?and lead author of the?NEJM?publication. Detailed outcomes from these studies were previously communicated in?May 2019. Data published in both?NEJM?and?The Lancet?contain additional analyses for these studies, including subgroup analyses, distribution of responses and safety. ?Following our recent?FDA?approval of TRIKAFTA in people ages 12 and older who have at least one?F508del?mutation, the simultaneous publication in?NEJM?and?The Lancet?and concurrent presentation at NACFC are further testament to the unprecedented results shown in these studies,? said?Reshma Kewalramani, M.D., Executive Vice President,?Global Medicines Developmentand Medical Affairs and Chief Medical Officer at Vertex. ?We have made significant progress toward bringing medicines targeting the underlying cause of disease to all people with CF, and we are grateful to all of the individuals and families who put their trust in us and participated in these studies.? The results were published online in conjunction with the presentation of both studies at the 33rd Annual?North American Cystic Fibrosis Conference?(NACFC),?October 31 through November 2in?Nashville. The oral presentation highlighting key outcomes from both studies is scheduled on?Friday, November 1?at?2:15 p.m. CT. These studies form the basis of the recent approval of TRIKAFTA by the U.S.?FDA?and support the Marketing Authorization Application (MAA) currently under review with the?European Medicines Agency. Other global submissions are also being prepared and will be submitted in 2020. Additional presentations at NACFC highlight data from across Vertex?s CF portfolio, including 96-week long-term safety and efficacy data for SYMDEKO??(tezacaftor/ivacaftor and ivacaftor) in patients ages?>12 years with CF homozygous for?F508del-CFTR?(F/F) or heterozygous for?F508del-CFTR?and a residual function mutation (F/RF), as well as data demonstrating the burden of illness in F/MF patients?>12 years of age.
Abstract Title |
Presentation Type |
Presenting Author |
Date/Time |
|
ELX/TEZ/IVA |
Phase 3 efficacy and safety of the ELX/TEZ/IVA triple combination in people with CF homozygous for the?F508delmutation |
Poster #508 |
Harry Heijerman |
Thursday, October 31 11:15 a.m. to 1:45 p.m. |
Phase 3 efficacy and safety of the ELX/TEZ/IVA triple combination in people with CF and?F508del/minimal function genotypes |
Poster #507 |
Raksha Jain |
Thursday, October 31 11:15 a.m. to 1:45 p.m. |
|
Phase 3 efficacy and safety of the ELX/TEZ/IVA combination therapy in people with CF homozygous for?F508del?or heterozygous for?F508del?and a minimal function mutation |
Oral Presentation: Workshop 17 |
Raksha Jain |
Friday, November 1 2:15 p.m. to 3:45 p.m. |
|
TEZ / IVA |
An open-label extension study of tezacaftor/ivacaftor (TEZ/IVA) therapy in patients aged?>12 years with cystic fibrosis (CF) homozygous for?F508del-CFTR?(F/F) or heterozygous for?F508del-CFTR?and a residual function mutation (F/RF) |
Late-Breaking Poster #853 |
Patrick Flume |
Thursday, October 31 11:15 a.m. to 1:45 p.m. |
CF Disease Burden |
Development of the cystic fibrosis questionnaire-revised preference based scoring algorithm (CFQ-R-8D) |
Poster #758 |
Lisa McGarry |
Thursday, October 31 11:15 a.m. to 1:45 p.m. |
Burden of illness in patients?>12 years with cystic fibrosis heterozygous for the?F508del?mutation and a minimal function mutation |
Poster #220 |
Greg Sawicki |
Thursday, October 31 11:15 a.m. to 1:45 p.m. |
View source version on businesswire.com:?https://www.businesswire.com/news/home/20191031005294/en/
Source:?Vertex Pharmaceuticals Incorporated Vertex Pharmaceuticals Incorporated? Investors:? Michael Partridge, 617-341-6108 or Leah Gibson, 617-961-1507 or Zach Barber, 617-341-6470 or Media:? mediainfo@vrtx.com? + 1-617-341-6992