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Health Canada Approves PrORKAMBI® (lumacaftor/ivacaftor) - the First Medicine to Treat the Underlying Cause of Cystic Fibrosis for People Ages 12 and Older with Two Copies of the F508del Mutation
-Approximately 1,500 people in
Cystic fibrosis is a rare, life-threatening genetic disease. People with
two copies of the F508del mutation represent the largest group of
people with CF. Of the approximately 4,000 people in
"With Health Canada's approval of ORKAMBI, people in
The approval of ORKAMBI was based on data from two Phase 3 studies (TRAFFIC and TRANSPORT) that enrolled more than 1,100 people with CF ages 12 and older with two copies of the F508del mutation.
Vertex continues to invest in CF research and development, including at
our
"Like PrKALYDECO® (ivacaftor) before it, ORKAMBI treats the
underlying defect in cystic fibrosis, but for a much larger part of the
CF population. Health Canada's approval of ORKAMBI is the first step in
getting this drug into the hands of the people who need it most, the
indicated patients for whom
About the Public Reimbursement Process in
Health
About CF and ORKAMBI
Cystic fibrosis is a rare genetic disease that is caused by defective or missing cystic fibrosis transmembrane conductance regulatory (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt and water into or out of the cell in a number of organs, including the lungs. 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. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.
ORKAMBI is a combination of lumacaftor, which is designed to increase the amount of protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. ORKAMBI is recommended to be taken every 12 hours - once in the morning and once in the evening.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR PrORKAMBITM (lumacaftor/ivacaftor)
ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 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. The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation.
IMPORTANT SAFETY INFORMATION
Before taking ORKAMBI, patients should tell their doctor if they have or have had liver problems or have kidney problems: have had an organ transplant; are using birth control (hormonal contraceptives, including oral, injectable, transdermal or implantable forms) because hormonal contraceptives should not be used as a method of birth control when taking ORKAMBI; are pregnant or plan to become pregnant because it is unknown if ORKAMBI will harm the unborn baby; are breastfeeding or planning to breastfeed as it is unknown if ORKAMBI passes into breast milk.
ORKAMBI can cause serious side effects including: High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving ORKAMBI. The patient's doctor will do blood tests to check their liver before they start ORKAMBI, every three months during the first year of taking ORKAMBI, and annually thereafter. The patient should call the 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 urine; confusion; or pale stools.
Respiratory events such as shortness of breath or chest tightness were observed in patients when starting ORKAMBI. If a patient has poor lung function, their doctor may monitor them more closely when starting ORKAMBI.
Increased blood pressure and decreased heart rate have been observed during treatment with ORKAMBI. Blood pressure should be monitored periodically in all patients during treatment. Caution should be observed in patients with pre-existing hypertension. Concomitant medications that result in an increase in blood pressure and/or a decrease in heart rate should be avoided to the extent possible during treatment with ORKAMBI.
ORKAMBI may affect the way other medicines work and other medicines may affect how ORKAMBI works. Therefore, the dose of ORKAMBI or other medicines may need to be adjusted when taken together. Patients should especially tell their doctor if they take: antifungal medicines, antibiotic medicines, anticonvulsant medicines, ranitidine, a medication used to treat peptic ulcers and gastroesophageal reflux disease, St. John's wort, benzodiazepines, antidepressants, anti-allergics, anti-inflammatories, immunosuppressants, cardiac glycosides, anticoagulants, glucocorticoids and proton pump inhibitors.
When taking ORKAMBI, a patient should tell their doctor if they stop ORKAMBI for more than 1 week as the doctor may need to change the dose of ORKAMBI or other medicines the patient is taking. It is unknown if ORKAMBI causes dizziness. A patient should not drive a car, use machinery, or do anything requiring alertness until the patient knows how ORKAMBI affects them.
Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ivacaftor, a component of ORKAMBI. The patient's doctor should perform eye examinations prior to and during treatment with ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include: shortness of breath and/or chest tightness; upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose; gastrointestinal symptoms including nausea, diarrhea, or gas; rash; fatigue; flu or flu-like symptoms; increase in muscle enzyme levels; and irregular, missed, or abnormal menstrual periods and heavier bleeding.
Please click here to see the full prescribing information for ORKAMBI.
Global Regulatory Submissions for ORKAMBI
ORKAMBI has received regulatory approval in
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, lead to CF by creating defective or too few CFTR proteins at the cell surface. The defective or missing CFTR protein results in poor flow of salt and water into or out of the cell in a number of organs, including 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 survival for Canadians with CF is currently estimated to be 51 years. Of the 40 people who died of CF in 2013 half were under the age of 35.
Collaborative History with
Vertex initiated its CF research program in 1998 as part of a
collaboration with CFFT, the nonprofit drug discovery and development
affiliate of the
About Vertex
Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research and
development sites and commercial offices in the United
States, Europe, Canada and
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements, as defined in
the Private Securities Litigation Reform Act of 1995, as amended,
including the quotes in the third and sixth paragraphs of this press
release and statements regarding Vertex's ongoing research and
development programs and the Canadian reimbursement process. While the
company believes the forward-looking statements contained in this press
release are accurate, there are a number of factors that could cause
actual events or results to differ materially from those indicated by
such forward-looking statements. Those risks and uncertainties include,
among other things, risks related to commercializing ORKAMBI in
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