"The European approval of KALYDECO is an important step in our commitment to bring transformative new medicines to people with cystic fibrosis," said
The European Commission's decision is based on positive findings from two global Phase 3 studies in which KALYDECO demonstrated significant and sustained improvements in breathing, weight gain and other measures of disease for people ages 6 and older with this specific genetic mutation, compared to placebo. In addition, people who took KALYDECO were 55 percent less likely to have pulmonary exacerbations, or periods of worsening in the signs and symptoms of the disease that often require treatment with antibiotics and hospital visits, than those who received placebo.
Fewer people in the KALYDECO treatment groups discontinued treatment due to adverse events than in the placebo groups. The majority of the adverse events associated with KALYDECO were mild to moderate. Adverse reactions very commonly observed in those taking KALYDECO (≥1/10) included headache; upper respiratory tract infection (common cold) including sore throat and nasal congestion; rash; diarrhoea; and abdominal pain (stomach ache). Two patients in the group receiving KALYDECO reported a serious adverse reaction of abdominal pain.
"Cystic fibrosis is a life-threatening genetic disease that causes devastating effects, particularly in the lungs, including the build up of thick, sticky mucus which becomes infected and severely limits normal breathing," said
"KALYDECO is an exciting new beginning in the treatment of cystic fibrosis, but we're not finished," said
KALYDECO was discovered as part of a collaboration with
KALYDECO™ (ivacaftor) is the first treatment to target 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 first approved by the
Vertex retains worldwide rights to develop and commercialize KALYDECO. KALYDECO is under Priority Review by the
Indication and Important Safety Information
KALYDECO (ivacaftor) is indicated for the treatment of cystic fibrosis (CF) in patients age 6 and older who have a G551D mutation in the CFTR gene.
KALYDECO is not effective in patients with CF who are homozygous for the F508del mutation in the CFTR gene. KALYDECO has not been studied in other populations of patients with CF.
Therefore, use of KALYDECO in these patients is not recommended.
KALYDECO is contraindicated in any patient with hypersensitivity to the active substance or to any of the excipients.
Moderate elevations in liver function tests (transaminases) are common in subjects with CF. Overall, the incidence and clinical features of transaminase elevations in clinical trials was similar between subjects in the ivacaftor and placebo treatment groups. In the subset of patients with a medical history of elevated transaminases, increases have been reported more frequently in patients receiving KALYDECO compared to placebo. Therefore, liver function tests are recommended prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. Patients who develop unexplained increased transaminase levels during treatment should be closely monitored until the abnormalities resolve and consideration should be given to the continuation of treatment after assessment of the individual benefits and risks.
Ivacaftor is a substrate of CYP3A4 and CYP3A5 isoenzymes. Medicinal products that inhibit or induce CYP3A activity, may impact the pharmacokinetics of ivacaftor. Ivacaftor is a weak CYP3A inhibitor and may modify the pharmacokinetics of medicinal products metabolised through the CYP3A system. In vitro studies indicated that ivacaftor has the potential to inhibit P-glycoprotein (P-gp) and CYP2C9. The dose of Kalydeco must be adjusted when concomitantly used with potent and moderate CYP3A inhibitors. Exposure to ivacaftor is reduced by the concomitant use of CYP3A inducers, therefore potentially resulting in loss of efficacy of KALYDECO.
The most common adverse reactions in patients treated with KALYDECO were abdominal pain (stomach ache), diarrhoea, dizziness, rash, upper respiratory tract reactions (including common cold, nasal congestion, redness of the throat, sore throat, runny nose, sinus congestion, and nose and throat inflammation), headache and bacteria in sputum. Two patients reported a serious adverse reaction of abdominal pain.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu once posted.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 70,000 people worldwide including 30,000 people in
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
Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.
Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, rheumatoid arthritis, epilepsy and other life-threatening diseases.
Founded more than 20 years ago 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, as amended, including without limitation, Dr. Leiden's statements in the second paragraph of this press release and Dr. Mueller's statements in sixth paragraph of this press release. Those risks and uncertainties include, among other things, risks related to the commercialization of KALYDECO and the development of additional medicines to treat cystic fibrosis and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the
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