Cell and gene therapies are in the limelight after a bit of a bumpy ride. Several marketing authorisations have been granted by health authorities around the world, but perhaps the ones attracting most attention are the first USA Food and Drug Administration (FDA) gene therapy approvals for chimeric antigen receptor T-cells (CAR-T), indicated for selected cancers of the haematopoietic system. The approved CAR-T drugs are CD19-directed genetically modified autologous T-cell immunotherapies. Novartis’ Kymriah (tisagenlecleucel) was approved on 30th August 2017, for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse and was the first ever US-approved gene therapy [1, 2]. Kite Pharma/Gilead’s Yescarta (axicabtagene ciloleucel) was approved on 18th October 2017, indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma [3]. Most recently, on 1st May 2018, Kymriah was also approved for the B-cell lymphoma indication [4]. Both drugs have been granted Priority Medicines (PRIME) regulatory support and are undergoing accelerated assessment by the European Medicines Agency (EMA).作者: StephenW 时间: 2018-7-3 20:57