FDA approves KALYDECO (ivacaftor) for use in infants with cystic fibrosis 12-24 months

recently, Vertex Pharmaceuticalsannounced that the U.SFood andDrug(
FDA(
) approved KALYDECO (ivacaftor) for use in infants with cystic fibrosis (CF) 12-24 months of agechildren based on clinical and/or in vitro assay data show that at least one mutation in their cystic fibrosis transmembrane conduction regulatory factor (CFTR) gene has a response to KALYDECOkalydeCO ® (ivacaftor)KALYDECO® (ivacaftor) is the first CF patient drug to treat CFTR gene-specific mutationsThe drug, known as the CFTR synergizer, is an oral drug designed to keep cfTR proteins open longer on the cell surface to improve the transport of salt and water on the cell membrane, thereby helping to hydrate and remove mucus from the airwaysKALYDECO HAS BEEN APPROVED IN THE UNITED STATES FOR THE TREATMENT OF CF PATIENTS 2 YEARS AND OLDER, AND ITS CLINICAL AND/OR IN VITRO ASSAY DATA SHOW THAT AT LEAST ONE OF THE 38 CFTR GENE MUTATIONS RESPONDS TO IVACAFTOR, FDA approval is based on data from the ongoing Phase 3 Open Label Safety Study, which studied 25 CFPATIENT children with cf-like mutations (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349DD, or 17H)studies have shown that safety is consistent with the safety previously observed in phase 3 studies in older children and adults;two patients had an increase of eight times the normal upper limit of liver enzymes, but continued to receive KALYDECO after the dose was interruptedadverse events
the most common adverse events (-30%) were cough (74%), fever (37%), elevated asperitase (37%), elevated acetaminophen (32%) and runny nose (32%) Two patients observed four severe adverse events 　　Vertex has also submitted a marketing authorization application to the European Medicines for use in infants aged 12-24 months and a decision is expected in the first half of 2019