Active Ingredient: Betibeglogene autotemcel
Betibeglogene autotemcel is indicated for the treatment of patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.
For this indication, competent medicine agencies globally authorize below treatments:
Intravenous
5,000,000 - 20,000,000 [CCID_50]
From 5,000,000 To 20,000,000 [CCID_50] once every day
The minimum recommended dose of betibeglogene autotemcel is 5.0 × 106 CD34+ cells/kg. In clinical studies doses up to 20 × 106 CD34+ cells/kg have been administered. The minimum recommended dose is the same for adults and adolescents 12 years of age and older.
Betibeglogene autotemcel is intended for autologous use and should only be administered once.
Patients are required to undergo HSC mobilisation followed by apheresis to obtain CD34+ stem cells which will be used for medicinal product manufacturing.
The minimum target number of CD34+ cells to be collected is 12 × 106 CD34+ cells/kg. If the minimum dose of betibeglogene autotemcel of 5.0 × 106 CD34+ cells/kg is not met after initial medicinal product manufacturing, the patient may undergo one or more additional cycles of mobilisation and apheresis, separated by at least 14 days, in order to obtain more cells for additional manufacture.
A back-up collection of CD34+ stem cells of ≥1.5 × 106 CD34+ cells/kg (if collected by apheresis) or >1.0 × 108 TNC/kg (if collected by bone marrow harvest) is required. These cells must be collected from the patient and be cryopreserved prior to myeloablative conditioning and infusion with betibeglogene autotemcel. The back-up collection may be needed for rescue treatment if there is: 1) compromise of betibeglogene autotemcel after initiation of myeloablative conditioning and before betibeglogene autotemcel infusion, 2) primary engraftment failure, or 3) loss of engraftment after infusion with betibeglogene autotemcel.
The treating physician should confirm that HSC transplantation is appropriate for the patient before myeloablative conditioning is initiated.
Full myeloablative conditioning must be administered before infusion of betibeglogene autotemcel. It is recommended that patients maintain haemoglobin (Hb) ≥11 g/dL for at least 30 days prior to mobilisation and during myeloablative conditioning. Iron chelation should be stopped at least 7 days prior to myeloablative conditioning. Prophylaxis for hepatic veno-occlusive disease (VOD) is recommended. Prophylaxis for seizures should be considered.
Myeloablative conditioning should not begin until the complete set of infusion bag(s) constituting the dose of betibeglogene autotemcel has been received and stored at the administration site, and the availability of the back-up collection is confirmed.
Any blood products required within the first 3 months after betibeglogene autotemcel infusion should be irradiated.
Restarting iron chelation after betibeglogene autotemcel infusion may be necessary and should be based on clinical practice. Phlebotomy can be used in lieu of iron chelation, when appropriate.
Betibeglogene autotemcel infusion should be completed as soon as possible and no more than 4 hours after thawing. Each infusion bag should be administered in less than 30 minutes. In the event that more than one infusion bag is provided, all infusion bags must be administered. The entire volume of each infusion bag should be infused.
Standard procedures for patient management after HSC transplantation should be followed after betibeglogene autotemcel infusion.
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