The new gene therapy option is available in Germany since April 2020. The orphan drug Zynteglo® is approved for the treatment of certain patients with the rare inherited disease β-thalassemia.
β-thalassemia is an inherited blood disorder caused by reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia to clinically asymptomatic individuals. The patients with manifesting disease are dependent on blood transfusions.
Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene†
) is indicated for the treatment of patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)–matched related HSC donor is not available.
Zynteglo is made individually for each patient from stem cells that are taken from their blood. Mobilization and apheresis, as well as chemotherapy, the so-called myeloablative conditioning with busulfan, must be carried out completely before administration. The new gene therapy is administered intravenously only once, with the dose depending on the patient's body weight.
Zyntelgo has received conditional approval. It is based on the data from the completed Phase I/II studies HGB-204 and HGB-205 as well as the data from the ongoing Phase III studies HGB-207 and HGB-212 and the long-term follow-up study LTF-303.
The full details in German can be found here
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