Active Ingredient: Elivaldogene autotemcel
Elivaldogene autotemcel is indicated for the treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling haematopoietic stem cell (HSC) donor is not available.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Intravenous, 5,000,000 cells elivaldogene autotemcel per kilogram of body weight, one dose.
Treatment consists of a single dose for infusion containing a dispersion of CD34+ cells in one or more infusion bags.
The minimum recommended dose of elivaldogene autotemcel is 5 × 106 CD34+ cells/kg. In clinical studies doses up to 38.2 × 106 CD34+ cells/kg have been administered.
Patients are required to undergo HSC mobilisation followed by apheresis to obtain CD34+ stem cells which will be used for medicinal product manufacturing.
The patient’s weight at first apheresis collection should be used to calculate the final dose.
The minimum target number of CD34+ cells to be collected is 12 × 106 CD34+ cells/kg. If the minimum dose of elivaldogene autotemcel 5 × 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 is required. These cells must be collected from the patient and be cryopreserved prior to initiating conditioning and infusion with elivaldogene autotemcel. The back-up collection may be needed for rescue treatment if there is: 1) compromise of elivaldogene autotemcel after initiation of conditioning and before elivaldogene autotemcel infusion, 2) primary engraftment failure, or 3) loss of engraftment after infusion with elivaldogene autotemcel.
The treating physician should confirm that elivaldogene autotemcel therapy is appropriate for the patient before conditioning is initiated.
Myeloablative conditioning must be administered before infusion of elivaldogene autotemcel.
Conditioning should not begin until the complete set of infusion bag(s) constituting the dose of elivaldogene autotemcel has been received at the administration site, and the availability of the back-up collection is confirmed.
Any blood products required within the first 3 months after elivaldogene autotemcel infusion should be irradiated.
After completion of the conditioning, there must be a minimum of 48 hours of washout before elivaldogene autotemcel infusion.
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