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    Home > Active Ingredient News > Drugs Articles > FDA approval of vertex cystic fibrosis targeting drug kalydeco for 2-5 years old children

    FDA approval of vertex cystic fibrosis targeting drug kalydeco for 2-5 years old children

    • Last Update: 2015-03-20
    • Source: Internet
    • Author: User
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    Source: Biovalley, March 19, 2015, vertex pharmaceutical recently got good news FDA has approved kalydeco (ivacftor) to carry one of 10 mutations of cystic fibrosis transmembrane conductance regulator (CFTR) (G551D, g1244e, g1349d, g178r, G551S, s1251n, s1255p, s549n, S549R, R117H) in children with cystic fibrosis (CF) Previously, the FDA had approved kalydeco for use in populations of patients 6 years and older with these mutations It is estimated that about 300 children aged 2-5 years old in the United States carry the above 10 mutations, of which about 150 carry R117H mutation and the other 150 carry other 9 mutations In the EU, kalydeco's application for expanded indications for 2-5-year-olds is under review It is estimated that there are more than 3400 patients suitable for kalydeco treatment in the United States, Canada, Europe and Australia However, it is worth mentioning that kalydeco is quite expensive, with a price of up to US $300000 / person / year, which also means that this expanded indication approval will generate nearly US $100 million of revenue for vertex every year Kalydeco's expanded indications were approved on the basis of an open label 24 week phase III clinical trial that investigated the safety and pharmacokinetics of a new formula based on body weight (50mg or 75mg twice daily) in 2-5-year-old children In order to meet the needs of pediatric patients in this age group, vertex has developed a new body weight based oral granule formula kalydeco (50mg and 75mg), which can be mixed with food or liquid for administration in patients who may not be able to swallow tablets Kalydeco is the first drug targeting at the root cause of cystic fibrosis (CF), which can make the defective CFTR protein play a normal function in patients The drug was first approved by FDA and EMA in 2012, and is used to treat patients aged 6 and over who have at least one copy of G551D mutation in CFTR gene In June 2014, the FDA further approved kalydeco for use in patients aged 6 years and over with CF carrying any of the nine non-g551d gated mutations In many countries around the world, kalydeco has been granted orphan drug status At present, vertex is rapidly promoting another very important combination therapy kalydeco / lumacafter for CF patients carrying f508del mutation At present, this combination therapy has achieved success in phase III clinical According to ISI group analysts, the total number of patients carrying f508del mutation is more than 28000 worldwide If the portfolio is finally approved for marketing, vertex will hold a super heavyweight drug comparable to Geely's hepatitis C star sovaldi Cystic fibrosis (CF) is a rare genetic disease, which is caused by the functional defect or deletion of CFTR protein caused by the mutation of CFTR gene CFTR proteins usually regulate the ion transport of cell membrane, and gene mutation can lead to the destruction or loss of protein product function When the ionic transport of cell membrane is interrupted, the viscosity of mucilage coating in some organs will thicken A major feature of the disease is the accumulation of thick mucus in the respiratory tract, which leads to dyspnea and repeated infection  
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