Children ages 2 to 5 with gating mutations in Australia to be provided immediate access to KALYDECO® (ivacaftor)

An agreement has been reached with the Australian Government to extend reimbursement for KALYDECO® (ivacaftor) to include children with cystic fibrosis (CF) ages 2 to 5 who have a G551D or other gating (Class III) mutation. 

The Department of Health can take up to three months to finalize the administrative arrangements to make medicines publicly available. Because early intervention is critical, Vertex will provide access to KALYDECO for all eligible children at no cost to the Australian Government between this announcement and the official Pharmaceutical Benefits Scheme (PBS) listing. 

“We know this agreement will be welcomed by parents, family members and friends who have been seeking this medicine for their children,” said Simon Bedson, Senior Vice President and International General Manager at Vertex. “We will be working closely with the CF clinics around Australia and hope to have KALYDECO available this week. We congratulate the new Minister, the PBAC, personnel from the Department of Health, MPs from all sides of the parliament, and the many Australians who have been seeking this outcome and have worked collaboratively towards it.”

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INDICATION FOR KALYDECO® (ivacaftor) IN AUSTRALIA

In Australia, KALYDECO (ivacaftor) is indicated for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have a G551D or other gating (class III) mutation in the CFTR gene. Please see the complete Australian Product Information that can be found on www.tga.gov.au.

 

INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO® (ivacaftor) IN THE UNITED STATES

KALYDECO (ivacaftor) 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 Prescribing Information for KALYDECO (ivacaftor).