Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference
- TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) real-world safety and effectiveness interim results show improved lung function and significant reductions in risk of pulmonary exacerbations, lung transplant and death for people with cystic fibrosis (CF) -
- Study in people with CF (F/F or F/MF genotypes) treated with TRIKAFTA® shows no mean loss of lung function after two years compared to those not treated with a CFTR modulator -
- Long-term real-world study results show significant benefits of initiating KALYDECO® (ivacaftor) at young age -
Additionally,
“These long-term and real-world studies show the potentially transformative benefits of treatment with CFTR modulators and add to the substantial body of evidence supporting treatment as early in life as possible,” said
Additional Presentations
In addition to the studies noted above, other
- Abstract WS08.04 — Results of real-world study in people with CF with select residual function mutations, treated with KALYDECO® (ivacaftor)
- Abstract WS17.02 — Results from an ORKAMBI® (lumacaftor/ivacaftor) exploratory phase 2 open-label extension study in children with CF ages 2-5
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 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 the 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 early 30s.
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 by binding to different sites on the CFTR protein. 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® is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 6 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 6 years of age.
Please see Important Safety Information below and [click here] for full
About KALYDECO® (ivacaftor)
In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane, which helps hydrate and clear mucus from the airways. KALYDECO® (ivacaftor) was the first medicine to treat the underlying cause of cystic fibrosis (CF) in people with specific mutations in the CFTR gene.
KALYDECO® is a prescription medicine used for the treatment of CF in patients aged 4 months and older who have at least one mutation in their CF 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 4 months of age.
Please see Important Safety Information below and [click here] for full
About ORKAMBI® (lumacaftor/ivacaftor)
In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little to no CFTR protein at the cell surface.
ORKAMBI® (lumacaftor/ivacaftor) is an oral medicine that is a combination of lumacaftor and ivacaftor. Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein. 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 lumacaftor and ivacaftor help hydrate and clear mucus from the airways.
ORKAMBI® is a prescription medicine used for the treatment of CF in patients age 2 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI® should only be used in these patients. It is not known if ORKAMBI® is safe and effective in patients under 2 years of age.
Please see Important Safety Information below and [click here] for full
IMPORTANT SAFETY INFORMATION for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), KALYDECO (ivacaftor), and ORKAMBI (lumacaftor/ivacaftor)
Patients should not take KALYDECO or TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; or St. John’s wort.
Patients should not take ORKAMBI if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; the seizure medicines phenobarbital, carbamazepine, or phenytoin; the sedatives and anti-anxiety medicines triazolam or midazolam; the immunosuppressant medicines cyclosporine, everolimus, sirolimus, or tacrolimus; or St. John’s wort.
Before taking KALYDECO, ORKAMBI, or TRIKAFTA patients should tell their doctor about all of their medical conditions, including if they: have or have had liver problems; have kidney problems; are pregnant or plan to become pregnant because it is not known if KALYDECO, ORKAMBI, or TRIKAFTA, will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if KALYDECO, ORKAMBI, or TRIKAFTA passes into breast milk. Before taking ORKAMBI, patients should tell their doctor if they have had an organ transplant, or if they are using a hormonal contraceptive including oral, injectable, transdermal, or implantable form as this should not be used as a method of birth control when taking ORKAMBI.
KALYDECO, ORKAMBI, or TRIKAFTA may affect the way other medicines work, and other medicines may affect how KALYDECO, ORKAMBI, or TRIKAFTA work. Therefore, the dose of KALYDECO, ORKAMBI, or TRIKAFTA may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.
KALYDECO or TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, use machinery, or do anything that needs them to be alert until they know how KALYDECO or TRIKAFTA affects them.
When taking ORKAMBI, patients should tell their doctor if they stop taking ORKAMBI for more than 1 week as their doctor may need to change the dose of ORKAMBI or other medicines the patient is taking.
Patients should avoid food or drink containing grapefruit while taking KALYDECO or TRIKAFTA.
KALYDECO, ORKAMBI, and TRIKAFTA can cause serious side effects, such as:
Liver damage and worsening of liver function in people taking TRIKAFTA with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease.
High liver enzymes in the blood have been reported in patients receiving KALYDECO, ORKAMBI, or TRIKAFTA. The patient's doctor will do blood tests to check their liver before starting treatment with KALYDECO, ORKAMBI, or TRIKAFTA; every 3 months during the first year of treatment; and every year while on treatment. 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.
Worsening of liver function in people with severe liver disease taking ORKAMBI. The worsening of liver function can be serious or cause death. Talk to your doctor if you have been told you have liver disease as your doctor may need to adjust the dose of ORKAMBI.
Breathing problems such as shortness of breath or chest tightness in patients when starting ORKAMBI, especially in patients who have poor lung function. If a patient has poor lung function, their doctor may monitor them more closely when starting ORKAMBI.
An increase in blood pressure in some people receiving ORKAMBI. The patient’s doctor should monitor their blood pressure during treatment with ORKAMBI.
Abnormality of the eye lens (cataract) in some children and adolescents treated with KALYDECO, ORKAMBI, or TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with KALYDECO, ORKAMBI, or TRIKAFTA to look for cataracts.
The most common side effects of KALYDECO 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.
The most common side effects of ORKAMBI include breathing problems, such as shortness of breath and chest tightness; nausea; diarrhea; fatigue; increase in a certain blood enzyme called creatinine phosphokinase; rash; gas; common cold, including sore throat, stuffy or runny nose; flu or flu-like symptoms; and irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding. Additional side effects seen in children include cough with sputum, stuffy nose, headache, stomach pain, and increase in sputum.
The most common side effects of TRIKAFTA include headache; diarrhea; upper respiratory tract infection (common cold), including stuffy and runny nose; stomach (abdominal) pain; inflamed sinuses; increase in liver enzymes; increase in a certain blood enzyme called creatine phosphokinase; rash; flu (influenza); and increase in blood bilirubin.
These are not all the possible side effects of KALYDECO, ORKAMBI, or TRIKAFTA. Please click product link to see the full
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