Ivacaftor Receives MAA Line Extension from the EMA for Children with CF


 Ivacaftor CAS No: 873054-44-5Known as the brand name Cystic Fibrosis drug Kalydeco, marketed by Vertex Pharmaceuticals, Ivacaftor recently received an MAA line extension. As of June 2015 children ages two through five who have specific mutations in the CFTR gene may be administered Ivacaftor as therapy. As a unique, tolerable and effectual treatment Ivacaftor acts as a novel cystic fibrosis transmembrane conductance regulator (CFTR) enhancer, which improves the pulmonary function in patients with Cystic Fibrosis and have the G551D CFTR-protein mutation. The use of Ivacaftor for children ages two through five, as well as children and adults ages six and older has already been approved by the FDA. Current statistics identify roughly 300 children between ages two and five diagnosed with one of the mutations of Cystic Fibrosis in America, with half of these patients possessing the gating defect in the CFTR protein and the other half having the R117H mutation of the disease.

“his study offered a new weight based formula of Ivacaftor made of oral granules which can be easily mixed with liquids or soft food to make the therapy more palatable.”

Successful phase 3 studies of Ivacaftor led to the FDA and EMA nods for an age extension. A 24 week study designed to evaluate the both the pharmokinetics and safety of Ivacaftor in children ages two through five was completed. This study offered a new weight based formula of Ivacaftor made of oral granules which can be easily mixed with liquids or soft food to make the therapy more palatable. The children studied were dispensed either 50 milligrams or 75 milligrams of Ivacaftor, based on weight, twice daily, exactly every 12 hours. The unique method of medication delivery made this option preferable for both parents and children, as many youngsters have difficulty swallowing pills or potent tasting liquid medications. The extended age range approval has now enabled over 3,400 more patients worldwide to receive Ivacaftor as a primary therapy for Cystic Fibrosis. Children and adults ages six and older are still regularly dispensed tablets of 150 milligrams. All patients taking Ivacaftor (Kalydeco) are prescribed this therapy to be taken alongside fat containing foods. The addition of high-fat content food with medication administration helps the absorption of Ivacaftor by the CFTR gene function effectively. This leads to not only increased weight gain for patients but also improved lung function. Commonly reported side effects include nasal congestion, minor rash, headache, dizziness and diarrhea. Ivacaftor has not been studied in children younger than two years of age. Patients taking Ivacaftor are also urged to avoid consumption of both grapefruit and Seville oranges during treatment to avoid contraindications.

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