Vertex has reached agreement with the French Economic Committee on Health Care Products (Comité Economique des Produits de Santé, or CEPS) for KALYDECO® (ivacaftor) for children with cystic fibrosis (CF) aged 2 years and older and weighing less than 25 kg who have one of nine approved gating mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D).
“Like other health authorities across Europe, the French Economic Committee recognizes the clinical benefit of providing early treatment with KALYDECO,” said Simon Bedson, Senior Vice President and International General Manager at Vertex. “This important step demonstrates our progress in getting our medicines to those who are eligible for them.”
The price and reimbursement approvals were published in the French Official Gazette on March 1, 2017, and KALYDECO granules will be available to all eligible people on the French National Health System four days later. Elsewhere in Europe, KALYDECO is available to people with CF aged 2 and older and weighing less than 25 kg who have one of nine approved gating mutations in Austria, Belgium, Denmark, England, Germany, the Netherlands, Norway, Scotland, Sweden, Switzerland and Wales.
In 2013, KALYDECO received the French “Prix Galien” award for the most promising rare disease medicine.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO® (ivacaftor) IN THE UNITED STATES
KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have one of the following mutations in their CF gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H. KALYDECO is not for use in people with CF due to other mutations in the CF gene. KALYDECO is not effective in patients with CF with two copies of the F508del mutation (F508del/F508del) in the CF gene. It is not known if KALYDECO is safe and effective in children under 2 years of age.
Patients should not take KALYDECO if they are taking certain medicines or herbal supplements such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. John's wort.
Before taking KALYDECO, patients should tell their doctor if they: have liver or kidney problems; drink grapefruit juice, or eat grapefruit or Seville oranges; are pregnant or plan to become pregnant because it is not known if KALYDECO will harm an unborn baby; and are breastfeeding or planning to breastfeed because is not known if KALYDECO passes into breast milk.
KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Therefore the dose of KALYDECO 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 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 affects them. Patients should avoid food containing grapefruit or Seville oranges while taking KALYDECO.
KALYDECO can cause serious side effects including:
High liver enzymes in the blood have been reported in patients receiving KALYDECO. The patient's doctor will do blood tests to check their liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while taking KALYDECO. 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.
Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. The patient's doctor should perform eye examinations prior to and during treatment with KALYDECO to look for cataracts. The most common side effects 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.
These are not all the possible side effects of KALYDECO. Please click here to see the full U.S. Prescribing Information for KALYDECO (ivacaftor).