– For the first time, approximately 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease–
-12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA-approved cystic fibrosis medicines are now also eligible for TRIKAFTA-
-Approval is based on two positive global Phase 3 studies in people with cystic fibrosis ages 12 and older with one F508del mutation and one minimal function mutation and in people with two F508del mutations-
BOSTON, MA, USA I October 21, 2019 I Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved 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. With this approval, for the first time, approximately 6,000 people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation (F/MF) have a medicine that targets the underlying cause of their CF. Additionally, approximately 12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA-approved CF medicines are now also eligible for TRIKAFTA.
“Today marks a milestone for CF patients, their families and Vertex. After a 20-year journey together, we have received FDA approval of TRIKAFTA: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future. For approximately 6,000 people with CF in the U.S., TRIKAFTA is the first medicine that can treat the underlying cause of their disease,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s Chairman, President and Chief Executive Officer. “I want to personally thank the hundreds of Vertex scientists who have been working on this program for nearly 20 years – many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.”
“Today’s approval is a historic moment in cystic fibrosis care, with the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease,” said Steven Rowe, M.D., Director, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham. “In clinical trials, TRIKAFTA was generally well tolerated and demonstrated improvements in multiple outcome measures in CF, including improvements in FEV1, improvements in respiratory symptoms and, in the 24-week F/MF study, a reduced rate of pulmonary exacerbations and improvements in BMI.”
“The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease,” said Reshma Kewalramani, M.D., Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “We remain committed to relentlessly pursuing the development of transformative therapies for all people living with this disease.”
Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen. Vertex is currently evaluating elexacaftor/tezacaftor/ivacaftor in people ages 6 through 11 with F/MF and F/F CF mutations in an ongoing Phase 3 study and is committed to evaluating elexacaftor/tezacaftor/ivacaftor in children <6 years of age as part of planned future studies.
For more information on Vertex’s patient assistance program, please visit VertexGPS.com.
About TRIKAFTA™
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine 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. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age. 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: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a 4-week Phase 3 study in 107 people with two F508del mutations (F/F).
Helping Patients Access TRIKAFTA™
The people who work at Vertex understand that medicines can only help people who can get them. The Vertex Guidance & Patient Support (Vertex GPS™) program provides a team of Vertex employees dedicated to helping eligible people in the United States who have been prescribed our medicines understand their insurance benefits and the resources that are available to help them.
Vertex also offers a co-pay assistance program for eligible people with commercial insurance coverage and a free medicine program for qualifying people who meet certain income and other eligibility criteria. More information is available by visiting www.VertexGPS.com or by calling 1-877-752-5933.
About Cystic Fibrosis
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. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, 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.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) – a rare, life-threatening genetic disease – and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.
Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters is now located in Boston’s Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry’s top places to work, including nine consecutive years on Science magazine’s Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
SOURCE: Vertex Pharmaceuticals
Post Views: 16
– For the first time, approximately 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease–
-12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA-approved cystic fibrosis medicines are now also eligible for TRIKAFTA-
-Approval is based on two positive global Phase 3 studies in people with cystic fibrosis ages 12 and older with one F508del mutation and one minimal function mutation and in people with two F508del mutations-
BOSTON, MA, USA I October 21, 2019 I Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) has approved 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. With this approval, for the first time, approximately 6,000 people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation (F/MF) have a medicine that targets the underlying cause of their CF. Additionally, approximately 12,000 people with one or two F508del mutations who are currently eligible for one of Vertex’s three other FDA-approved CF medicines are now also eligible for TRIKAFTA.
“Today marks a milestone for CF patients, their families and Vertex. After a 20-year journey together, we have received FDA approval of TRIKAFTA: a single breakthrough medicine with the potential to treat up to 90% of all people with CF in the future. For approximately 6,000 people with CF in the U.S., TRIKAFTA is the first medicine that can treat the underlying cause of their disease,” said Jeffrey Leiden, M.D., Ph.D., Vertex’s Chairman, President and Chief Executive Officer. “I want to personally thank the hundreds of Vertex scientists who have been working on this program for nearly 20 years – many of whom have dedicated their entire careers to changing the course of this disease; the CF Foundation which has provided support, encouragement and help throughout the journey; and most importantly the thousands of patients, caregivers, doctors and advocates who have courageously and persistently worked side-by-side with us to get to where we are today.”
“Today’s approval is a historic moment in cystic fibrosis care, with the potential for more people to benefit from CFTR modulator therapy to treat the basic defect of their disease,” said Steven Rowe, M.D., Director, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham. “In clinical trials, TRIKAFTA was generally well tolerated and demonstrated improvements in multiple outcome measures in CF, including improvements in FEV1, improvements in respiratory symptoms and, in the 24-week F/MF study, a reduced rate of pulmonary exacerbations and improvements in BMI.”
“The incredible speed of this approval underscores our shared sense of urgency with the FDA and the CF community for bringing this medicine to eligible people with CF, particularly those without a medicine targeting the underlying cause of their disease,” said Reshma Kewalramani, M.D., Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “We remain committed to relentlessly pursuing the development of transformative therapies for all people living with this disease.”
Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen. Vertex is currently evaluating elexacaftor/tezacaftor/ivacaftor in people ages 6 through 11 with F/MF and F/F CF mutations in an ongoing Phase 3 study and is committed to evaluating elexacaftor/tezacaftor/ivacaftor in children <6 years of age as part of planned future studies.
For more information on Vertex’s patient assistance program, please visit VertexGPS.com.
About TRIKAFTA™
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine 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. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age. 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: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a 4-week Phase 3 study in 107 people with two F508del mutations (F/F).
Helping Patients Access TRIKAFTA™
The people who work at Vertex understand that medicines can only help people who can get them. The Vertex Guidance & Patient Support (Vertex GPS™) program provides a team of Vertex employees dedicated to helping eligible people in the United States who have been prescribed our medicines understand their insurance benefits and the resources that are available to help them.
Vertex also offers a co-pay assistance program for eligible people with commercial insurance coverage and a free medicine program for qualifying people who meet certain income and other eligibility criteria. More information is available by visiting www.VertexGPS.com or by calling 1-877-752-5933.
About Cystic Fibrosis
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. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, 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.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) – a rare, life-threatening genetic disease – and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.
Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters is now located in Boston’s Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry’s top places to work, including nine consecutive years on Science magazine’s Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
SOURCE: Vertex Pharmaceuticals
Post Views: 16
