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Vertex Receives Australian Approval for KALYDECO™ (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People with a Specific Genetic Mutation (G551D)
-- Approximately 250 people in
"The approval of KALYDECO in
The approval of KALYDECO was based on data from two global Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function and weight gain compared to those who received placebo. In one study, adults and adolescents who took KALYDECO were also significantly less likely to experience pulmonary exacerbations, which are periods of worsening respiratory signs and symptoms that require increased treatment with antibiotics and hospital visits.
The most common serious adverse events reported in Phase 3 studies included abdominal pain, increased liver enzymes and low blood sugar, which occurred in less than 1 percent of patients. Adverse events commonly observed in those taking KALYDECO included headache, upper respiratory tract infection (common cold), stomach pain and diarrhea. Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo group. The majority of adverse events associated with KALYDECO were mild to moderate.
KALYDECO was discovered by Vertex as part of a collaboration with
About the Australian Funding Process
Australian approval and reimbursement of a new medicine is a multi-step
process. Once a new medicine receives approval from the TGA, it is
assessed for effectiveness and cost-effectiveness by the
PBS Information: KALYDECO is not listed on the
About Cystic Fibrosis1
Cystic fibrosis is a rare, life-threatening genetic disease affecting
approximately 70,000 people worldwide, including 3,000 people in
CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (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 more than 1,800 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic or genotyping test, lead to CF by creating non-working or too few CFTR protein channels at the cell surface. The absence of working CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs. As a result, thick, sticky mucus builds up and blocks the passages in many organs, particularly in the lungs, leading to a variety of symptoms, including chronic lung inflammation, recurrent infections and progressive lung damage. The most common cause of death among people with CF is lung disease, which results from recurring infections and chronic lung inflammation.
For more than 15 years, Vertex has been working to develop new medicines to treat the underlying cause of CF in as many people as possible.
One in 2,500 babies in
Chronic antibiotic treatment is widespread among Australians with CF regardless of age. In 2011, 90 percent of the youngest patients, those under 2 years, received treatment with antibiotics, and at least 94 percent of those in all other age groups also received antibiotic treatment. Nearly half (47 percent) of Australians with CF were hospitalised at least once in 2011. Of these patients, approximately half (49 percent) spent at least 14 days in the hospital.
KALYDECO™ (ivacaftor) is the first medicine to treat the underlying
cause of CF in people with the G551D mutation in the CFTR gene.
Known as a CFTR potentiator, KALYDECO is an oral medicine that aims to
help the CFTR protein function more normally once it reaches the cell
surface, to help hydrate and clear mucus from the airways. KALYDECO
(150mg, q12h) was approved by the
Vertex retains worldwide rights to develop and commercialise KALYDECO.
Indication and Important Safety Information for KALYDECO™ (ivacaftor)
KALYDECO (150mg tablets) is indicated for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have a G551D mutation in the CFTR gene.
KALYDECO is not for use in people with CF due to other mutations in the CFTR gene. It is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CFTR gene. The efficacy and safety of KALYDECO in children younger than 6 years of age have not been evaluated.
High liver enzymes (transaminases, ALT and AST) have been reported in patients receiving KALYDECO. It is recommended that ALT and AST be assessed prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. Patients who develop increased transaminase levels should be closely monitored until the abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal. Following resolution of transaminase elevations, consider the benefits and risks of resuming KALYDECO dosing. Moderate transaminase elevations are common in subjects with CF. Overall, the incidence and clinical features of transaminase elevations in clinical trials was similar between subjects in the KALYDECO and placebo treatment groups. In the subset of patients with a medical history of elevated transaminases, increased ALT or AST have been reported more frequently in patients receiving KALYDECO compared to placebo.
Use of KALYDECO with medicines that are strong CYP3A inducers such as the antibiotics rifampin and rifabutin; seizure medications (phenobarbital, carbamazepine, or phenytoin); and the herbal supplement St. John's Wort substantially decreases exposure of KALYDECO, which may diminish effectiveness. Therefore, co-administration is not recommended.
The dose of KALYDECO must be adjusted when concomitantly used with potent and moderate CYP3A inhibitors. The dose of KALYDECO must be adjusted when used in patients with moderate or severe hepatic disease.
KALYDECO can cause serious adverse reactions including abdominal pain and high liver enzymes in the blood. The most common side effects associated with KALYDECO include headache; upper respiratory tract infection (the common cold), including sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; and dizziness. These are not all the possible side effects of KALYDECO. A list of the adverse reactions can be found in the full product labeling for each country where KALYDECO is approved. Patients should tell their healthcare providers about any side effect that bothers them or doesn't go away.
For more information, please see the Consumer Medical Information and Product Information at http://bit.ly/18wlMld.
Vertex is a global biotechnology company that aims to discover, develop and commercialise innovative new medicines so people with serious diseases can lead better lives. Vertex scientists and the company's collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis and other life-shortening diseases.
Founded in 1989, Vertex now employs more than 2,200 people at sites
around the world. Headquartered in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, statements regarding the Australian approval and
reimbursement process. While Vertex 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, the risks
listed under Risk Factors in Vertex's annual report and quarterly
reports filed with the
VRTX — GEN
1 Cystic Fibrosis Australia. Cystic Fibrosis in
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