New drug for cystic fibrosis! Vertex drug kalydeco is approved by EU for 6-12 months

December 13, 2019 / BIOON / -- vertex pharmaceutical company is a global leader in the treatment of cystic fibrosis (CF) Recently, the company announced that the European Commission (EC) has approved the expansion of kalydeco (ivacftor) label to include children with cystic fibrosis (CF) who have one of the following nine mutations in CFTR gene from 6 to 12 months: G551D, g1244e, g1349d, g178r, G551S, s1251n, s1255p, s549n, S549R This approval makes kalydeco the first and only drug in Europe that can be used to treat the internal causes of CF patients with specific mutations in the CFTR gene within 6 to 12 months In the European Union, kalydeco has been approved before: (1) for the treatment of CF patients with one of nine mutations (G551D, g1244e, g1349d, g178r, G551S, s1251n, s1255p, s549n, S549R) in CFTR gene for 12 months or more (2) It is used to treat CF adults aged 18 and over who have R117H mutation in CFTR gene The approval is based on data from an ongoing open label phase III study (arrival) Data show that the safety observed by Kalydeco in CFTR infants between 6 and 12 months of age with a specific mutation in the CF gene is consistent with previous phase III studies in older pediatric and adult patients, and shows improvement in sweat chloride, a key secondary end point of the study Kalydeco is the first drug to target the underlying cause of cystic fibrosis (CF), which can make the defective CFTR protein in patients function normally 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 It is worth mentioning that in October this year, vertex's breakthrough triple therapy for cystic fibrosis, trikafta, was approved by the US FDA for use in CF patients with at least one f508del mutation and age ≥ 12 years old in CFTR gene, specifically: Patients with one f508del mutation and one minimal functional mutation, or patients with two f508del mutations Prior to that, FDA has granted trikafta priority review qualification, fast track qualification and breakthrough drug qualification (BTD) At present, vertex's CF drugs on the market include kalydeco (ivacftor), orkabi (lumacaftor / ivacftor) and symkevi / symdeko (tezacaftor / ivacftor) These three drugs can treat about 40000 patients worldwide, accounting for about 50% of all CF patients The new triple therapy approved this time, trikafta, can expand the scope of treatment to 90% of CF patients in the world (biology Com) original source: vertex announcements European Commission approval for kalydeco ® (ivacftor) in ants with cyclic fibrosis ages 6 months to less than 12 months with certificate mutations in the CFTR gene