EU approves expanded indications Kalydeco orphan drug price

The European Commission (EC) has approved the expansion of orphan drug Kalydeco (ivacaftor) indications, for carrying the cystic fibrosis transmembrane conductance regulator (CFTR) 9 种 gated one mutation (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R) of 2-5-year-old cystic fibrosis (CF) in pediatric patients, as well as carrying the R117H mutation of 18 years and older adult CF patients. Previously, Kalydeco been approved for the EU to carry nine kinds gated one mutation in CF patients 6 years and older. In Europe, about 125 cases of children 2-5 years old CF patients carry mutations in one of the gated above nine kinds, about 350 cases of adult CF patients carrying the R117H mutation. It is worth mentioning that, Kalydeco quite expensive, priced up to $ 300,000 / person / year, which means that the approval of this expanded indication, will add nearly $ 140 million in revenue annually Vertex.
Kalydeco expand pediatric indication is based on an open-label Phase III clinical trials 24 weeks, the study looked at the weight-based dosing regimens new formula safe for children 2-5 years old patients (50mg or 75mg twice daily) and pharmacokinetics. To meet this age group needs of pediatric patients, Vertex has developed a new weight-based Kalydeco oral granular formulations (50mg and 75mg), patients may be unable to swallow tablets, the drug can be mixed with food or liquid to medicine. Kalydeco expand adult indication is based on positive data from a Phase III study in 69 cases carrying the R117H mutation in adult CF patients conducted.
In many countries, Kalydeco were granted orphan drug status, the drug is the first targeted therapy for cystic fibrosis (CF) the underlying cause, can make the patient the defective CFTR protein function properly, the drug in 2012 It was first eligible for FDA and EU approval for the treatment of CFTR gene has at least one copy of the G551D mutation in 6 years and older with cystic fibrosis (CF) patients. After, Kalydeco indications continue to be expanded, but also for the Vertex company a big moneymaker.