GSK gene therapy strimvelis is approved to be listed in Europe, promising an invalid refund

Source: 36 krypton 2016-08-11 recently, strimvelis, a gene therapy developed by Italian scientists and British pharmaceutical giant GSK, was approved by the European Commission (EC) for the treatment of pediatric patients with ADA-SCID According to MIT Technology Review, GSK announced last week that the price of strimvelis therapy was 594000 euros, becoming one of the most expensive one-time therapies in history, and promised an invalid refund It is understood that ADA-SCID is a very rare disease Newborns with this disease cannot produce a type of white blood cells, do not have a healthy and sound immune system function, resulting in loss of resistance to daily infection, and must live in a sterile environment It is estimated that about 15 patients in Europe are diagnosed with the disease every year, and such patients generally cannot live to 2 years old unless their immune system is restored through a matched bone marrow transplant The difference between strimvelis gene therapy and the previous treatment is that it can be 100% cured by gene repair This therapy is the first one to achieve the effect of life-long cure and commercialization of gene therapy Strimvelis therapy is to take stem cells from the patient's bone marrow, transfer a normal copy of ADA gene into the stem cells, and then take intravenous infusion to reintroduce the stem cells into the patient's body, and then some of the stem cells will nest in the bone marrow The treatment has been carried out in 18 children, the first being 15 years ago At present, the 18 patients with ADA-SCID treated by strimvelis are still alive A key study showed that all pediatric ADA-SCID patients had a 3-year survival rate of 100% after treatment with strimvelis Strimvelis only needs to be administered once, and does not rely on third-party donors There is no risk of immune incompatibility caused by rejection Currently, the cost of another therapy for ADA-SCID is about 900000 euros The price of strimvelis is two-thirds of that GSK said patients receiving its gene therapy will be registered for follow-up and guaranteed an invalid refund According to the accumulated experience, GSK has a refund probability of about one sixth In addition, GSK has indicated that installment payments are acceptable