Vertex to Present New Data at European and North American Virtual Cystic Fibrosis Conferences Highlighting Long-Term Use of CFTR Modulators
- Oral presentation of interim results from TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) open-label safety extension study to be presented at the
- Six presentations highlighting data from KALYDECO® (ivacaftor), ORKAMBI® (lumacaftor/ivacaftor) and TRIKAFTA to be presented at
“As we continue to reach additional people with CF with our medicines, gaining a better understanding of their long-term and real-world impact becomes even more important,” said
Data highlighting interim results from the ongoing TRIKAFTA open-label extension (OLE) study to evaluate long-term safety and efficacy in people with CF ages 12 and older with F508del/Minimal Function (F/MF) or F508del/F508del (F/F) genotypes who completed pivotal studies will be presented at the
A full listing of
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ELX/TEZ/IVA |
A phase 3, open-label extension study of elexacaftor/tezacaftor/ivacaftor: interim analysis of safety and efficacy in people with cystic fibrosis and F508del/minimal function or F508del/F508del genotypes |
ECFS Oral Presentation |
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Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis heterozygous for F508del and a minimal function mutation: results from a phase 3 clinical study |
ECFS published abstract: |
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NACFC Poster Presentation #447 |
Professor Fajac |
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Impact of elexacaftor/tezacaftor/ivacaftor triple combination therapy on health-related quality of life in people with cystic fibrosis homozygous for F508del: results from a phase 3 clinical study |
ECFS published abstract: |
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NACFC Poster Presentation #478 |
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IVA |
An observational study of ivacaftor in people with cystic fibrosis and selected non-G551D gating mutations: outcomes from the third interim analysis of the VOCAL study |
NACFC Poster Presentation #466 |
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Ivacaftor in 4 to < 6-month-old infants with a gating mutation: results of a 2-part, single-arm, phase 3 study |
NACFC Poster Presentation #415 |
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Real-world outcomes in children aged 2-5 with CF treated with ivacaftor |
NACFC Poster Presentation #141 |
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LUM/IVA |
Long-term safety of lumacaftor/ivacaftor in persons with cystic fibrosis aged 2-5 years homozygous for the F508del-CFTR mutation (F/F) |
ECFS Published abstract:
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Disease progression in F508del homozygous (F/F) persons with cystic fibrosis treated with lumacaftor/ivacaftor (LUM/IVA): interim results of a long-term safety study using data from the US Cystic Fibrosis Foundation Patient Registry (CFFPR) |
ECFS Published abstract:
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NACFC Poster Presentation #190 |
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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.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO® (ivacaftor), TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), and ORKAMBI® (lumacaftor/ivacaftor)
What is KALYDECO?
KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 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 6 months of age.
What is TRIKAFTA?
TRIKAFTA is a prescription medicine used for the treatment of CF in patients aged 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.
What is ORKAMBI?
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.
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, TRIKAFTA, or ORKAMBI, patients should tell their doctor about all of their medical conditions, including if they: have or have had liver problems; have kidney problems; have had an organ transplant; are pregnant or plan to become pregnant because it is not known if KALYDECO, TRIKAFTA, or ORKAMBI will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if KALYDECO, TRIKAFTA, or ORKAMBI passes into breast milk. Before taking ORKAMBI, patients should tell their doctor if they are using birth control as hormonal contraceptives, including oral, injectable, transdermal, or implantable forms should not be used as a method of birth control when taking ORKAMBI.
KALYDECO, TRIKAFTA, or ORKAMBI may affect the way other medicines work, and other medicines may affect how KALYDECO, TRIKAFTA, or ORKAMBI work. Therefore, the dose of KALYDECO, TRIKAFTA, or ORKAMBI 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 or
KALYDECO, TRIKAFTA, and ORKAMBI can cause serious side effects, such as:
High liver enzymes in the blood have been reported in patients receiving KALYDECO, TRIKAFTA, or ORKAMBI. The patient's doctor will do blood tests to check their liver before starting treatment with KALYDECO, TRIKAFTA, or ORKAMBI; 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.
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, TRIKAFTA, or ORKAMBI. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with KALYDECO, TRIKAFTA, or ORKAMBI 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 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.
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.
These are not all the possible side effects of KALYDECO, TRIKAFTA, or ORKAMBI. Please click product link to see the full Prescribing Information for KALYDECO, TRIKAFTA, or ORKAMBI.
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