Table. Gene therapies licensed for use in Europe.
|
Product Year of approval |
Indication*1 | Prevalence*2 | Viral vector or cell therapy | Application |
| Alipogentiparvovec(Glybera) 2012 (approval expired in 2017) |
Adults with familial lipoprotein lipase deficiency (LPLD) with severe or multiple episodes of pancreatitis in spite of a low-fat diet | Target population in Germany: 17–35 | AAV1 vector coding for human LPL gene | Several intramuscular injections of the vector in one session |
| Talimogen laherparepvec (Imlygic) 2015 |
Adults with non-resectable, local or distant metastatic melanoma (stages IIIB, IIIC, and IVM1a) | Target population in Germany: 75–225 | Vector coding for human GM-CSF, which is based on attenuated HSV-1 | Repeated injection of the vector into cutaneous, subcutaneous, and/or nodal lesions |
| Strimvelis 2016 |
Patients with severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID), for which no suitable human leukocyte antigen (HLA) compatible stem cell donor from the family is available | Prevalence 0.02 : 10 000*3 means a prevalence for all of Germany of about 160 | Ex vivo therapy of autologous CD34+-cells transduced by using retroviral vector carrying the ADA coding sequence | Intravenous administration of the genetically modified CD34+ –cells |
| Tisagenlecleucel (Kymriah) 2018 |
Patients aged up to 25 years with refractory or recurrent B-cell acute lymphoblastic leukemia (ALL); adults with diffuse large B cell lymphoma (DLBCL) after a minimum of two systemic treatments | Target population in Germany: 438–702 | Ex vivo therapy of lentivirally transduced autologous T-cells that express a chimeric antigen receptor (CAR) directed against CD19 | Intravenous administration of the genetically modified T-cells |
| Axicabtagene ciloleucel (Yescarta) 2018 |
Adults with recurrent or refractory diffuse large B cell lymphoma (DLBCL) and primary mediastinal large B cell lymphoma (PMBCL) after a minimum of two systemic treatments | Target population in Germany: 871–1 064 | Ex vivo therapy of retrovirally transduced autologous T-cells, that express a chimeric antigen receptor (CAR) directed against CD19 | Intravenous administration of the genetically modified T-cells |
| Voretigen neparvovec (Luxturna) 2018 |
Patients with vision loss owing to hereditary retinal dystrophy, as a result of confirmed biallelic RPE65 mutations | Target population in Germany: 189–290 | AAV2 vector coding for the human RPE65 gene | Subretinal injection of the vector |
| Betibeglogene autotemcel (Zynteglo) 2019 |
Patients > 12 years with transfusion dependent β-thalassemia (TDT), who do not have a β0/β0 genotype, and for whom no stem cell donor is available | Target population in Germany: 49–53 | Ex vivo therapy of CD34+-cells transduced with a lentiviral vector coding for β-Globin (T87Q) | Intravenous administration of the genetically modified CD34+ -cells |
| Onasemnogene abeparvovec (Zolgensma) 2020 |
Patients with spinal muscular atrophy (SMA) owing to a biallelic mutation in the SMN1 gene and the clinical phenotype of SMA type 1 or up to 3 SMN2 copies | Target population in Germany: 71–120 patients with SMA type 1, 770 bis 941 patients with later disease onset, SMA incidence at birth about 100 per year | AAV9 vector coding for the human SMN1- gene | Intravenous infusion of the vector |
In contrast to the usual practice of the Deutsches Ärzteblatt we list the medications used for the therapy under their proprietary names in order to facilitate easier recognition, as even experts are often unfamiliar with the vector names. See Box 2 regarding the terminology of gene therapeutics. *1 Details on the precise area of use should be taken from the product leaflets/information. *2 The reported prevalence rates of patients suitable for the relevant therapy are often subject to great uncertainties. Unless reported otherwise the data are from the reports of the Institute for Quality and Efficiency in Healthcare (IQWiG) in the context of benefit assessments by the Federal Joint Committee (www.g-ba.de). Some prevalence rates refer to patients in the statutory health insurance scheme (GKV), this proportion accounts for 90 % of the total population. *3 vgl. www.ema.europa.eu/en/medicines/human/orphan-designations/eu305313