Vertex Highlights First Oral Presentation of Phase 3 Clinical Data of the Vanza Triple and New Data on Long-Term Impact of TRIKAFTA® at the North American Cystic Fibrosis Conference
– Phase 3 data on investigational vanza triple demonstrates non-inferiority to TRIKAFTA ® in ppFEV1 and further improvement of CFTR function as measured by sweat chloride –
– Real-world evidence and clinical studies of TRIKAFTA ® continue to show sustained long-term benefits including improvement in pancreatic function in young patients –
“TRIKAFTA has transformed the treatment of CF in the 5 years since its approval, changing the outlook for patients with CF,” said
Data on the Investigational Once Daily Next-in-Class Triple Combination Therapy, vanzacaftor/tezacaftor/deutivacaftor
“The additional, significant reductions in sweat chloride we see in the vanza triple clinical trials are noteworthy. I believe this improvement in CFTR function may lead to important benefits for people with CF,” said
Data from the Phase 3 clinical studies of the vanza triple will be presented in the following sessions:
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“Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Adolescents and Adults with CF: Results from Two Randomized, Active-Controlled Phase 3 Trials,” will be an oral presentation during a session entitled: “Cutting Edge Research: What’s New” on
Friday, September 27 , from10:15 a.m. to 12:15 p.m. EDT and be a poster presentation onFriday, September 27 , from1:15-2:15 p.m. EDT . -
“Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Children 6 Through 11 Years of Age with Cystic Fibrosis,”will be a poster presentation on
Friday, September 27 , from1:15-2:15 p.m. EDT .
The vanza triple was granted Fast Track and Orphan Drug Designations from the
Long-Term Benefits of TRIKAFTA ®
“I have seen first-hand the positive long-term impact that improvement of CFTR function by TRIKAFTA can have on patients’ clinical outcomes. These improvements are particularly striking for me as a physician caring for young children, where improvements in things like lung function, pancreatic function and quality of life are so meaningful,” said Professor
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“Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children 2 years and older with cystic fibrosis and at least one F508del allele: 96-week results from an open-label extension study,”will be a posterpresentation on
Friday, September 27 , at12:15-1:15 p.m. EDT . -
“Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children 6 years and older with cystic fibrosis and at least one F508del allele: Results from a 192-week extension study,” will be a posterpresentation on
Friday, September 27 , at1:15-2:15 p.m. EDT . -
“Effectiveness of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in people with cystic fibrosis and non-F508del CFTR variants: Interim results from a registry-based study,” will be a poster presentation on
Friday, September 27 , from12:15-1:15 p.m. EDT . -
“Long-term benefits of early ivacaftor (IVA) initiation in people with cystic fibrosis (CF),” will be a poster presentation on
Friday, September 27 , from1:15-2:15 p.m. EDT .
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands 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, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein 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, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.
Learn more about the importance of sweat chloride (SwCl) in cystic fibrosis.
Today
About vanzacaftor/tezacaftor/deutivacaftor (the “vanza triple”)
In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane.
Investigational vanzacaftor/tezacaftor/deutivacaftor was granted Fast Track and Orphan Drug Designations from the
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways.
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation, or another mutation responsive to TRIKAFTA®, in the 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 2 years of age.
TRIKAFTA
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. 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 2 years of age.
IMPORTANT SAFETY INFORMATION
Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: are allergic to TRIKAFTA or any ingredients in TRIKAFTA, have kidney problems,have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk.
Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works.The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin.
Patients should avoid food or drink that contains grapefruit while taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
Liver damage and worsening of liver function in patients with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in patients without liver disease.
High liver enzymes in the blood, which is a common side effect in patients treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patient’s doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine.
Serious allergic reactions have happened to patients who are treated with TRIKAFTA. Call your healthcare provider or go to the emergency room right away if you have any symptoms of an allergic reaction. Symptoms of an allergic reaction may include: rash or hives; tightness of the chest or throat or difficulty breathing; swelling of the face, lips and/or tongue; difficulty swallowing; and light-headedness or dizziness.
Abnormality of the eye lens (cataract) has been noted in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts.
The most common side effects of TRIKAFTA include headache, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, flu (influenza), inflamed sinuses, and increase in blood bilirubin.
Patients should tell their doctor if they have any side effect that bothers them or that does not go away. These are not all the possible side effects of TRIKAFTA.For more information, patients should ask their doctor or pharmacist.
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KALYDECO®
INDICATIONS AND USAGE
KALYDECO (ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 1 month of age.
IMPORTANT SAFETY INFORMATION
Before taking KALYDECO, patients should tell their doctor about all their medical conditions, including if they: have liver or kidney problems; are allergic to KALYDECO or any ingredients; are pregnant or plan to become pregnant because it is not known if KALYDECO will harm an unborn baby; and are breastfeeding or planning to breastfeed because is not known if KALYDECO passes into breast milk.
Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take the antibiotics rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.
KALYDECO can cause dizziness in some patients who take it. If patients experience dizziness, they should not drive or operate machines until symptoms improve.
Patients should avoid food or drink containing grapefruit while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood, which have happened in patients receiving KALYDECO. The patient’s doctor will do blood tests to check their liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while taking KALYDECO. For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.
Serious allergic reactions have happened to patients who are treated with KALYDECO. Patients should call their healthcare provider or go to the emergency room right away if they have symptoms of an allergic reaction. Symptoms of an allergic reaction may include rash or hives, tightness of the chest or throat or difficulty breathing, and light-headedness or dizziness.
Abnormality of the eye lens (cataract), which has happened in some children and adolescents receiving KALYDECO. The patient’s doctor should perform eye examinations before and during treatment with KALYDECO to look for cataracts.
The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.
Use of KALYDECO in patients aged 1 month to less than 6 months born from a pregnancy lasting (gestational age) less than 37 weeks has not been evaluated.
These are not all the possible side effects of KALYDECO. Please click here to see the full Prescribing Information for KALYDECO.
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