Source: European Medicines Agency (EU) Revision Year: 2023 Publisher: Orchard Therapeutics (Netherlands) B.V., Basisweg 10, 1043 AP Amsterdam, The Netherlands
Libmeldy 2-10 × 106 cells/mL dispersion for infusion.
| Pharmaceutical Form |
|---|
|
Dispersion for infusion. A clear to slightly cloudy, colourless to yellow or pink dispersion. |
Libmeldy (atidarsagene autotemcel) is a genetically modified autologous CD34+ cells enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector expressing the human arylsulfatase A (ARSA) gene.
Each patient-specific infusion bag of Libmeldy contains atidarsagene autotemcel at a batch-dependent concentration of genetically modified autologous CD34+ cells enriched population. The medicinal product is packaged in one or more infusion bags overall containing a dispersion of 2-10 x106 cells/mL of viable CD34+ cells enriched population suspended in a cryopreservative solution.
Each infusion bag contains 10 to 20 mL of Libmeldy.
The quantitative information of medicinal product, including the number of infusion bags (see section 6) to be administered, is presented in the Lot Information Sheet located inside the lid of the cryoshipper used for transport.
Excipients with known effect: This medicinal product contains 3.5 mg sodium per mL and 55 mg dimethylsulfoxide (DMSO) per mL.
For the full list of excipients, see section 6.1.
| Active Ingredient | Description | |
|---|---|---|
| Atidarsagene autotemcel |
Atidarsagene autotemcel is an ex vivo genetically modified autologous CD34+ hematopoietic stem and progenitor cell (HSPC) gene therapy. Autologous CD34+ HSPCs are collected from patient bone marrow (BM) harvest or from mobilised peripheral blood (mPB) and transduced with a lentiviral vector (ARSA LVV), which inserts one or more copies of the human ARSA complementary deoxyribonucleic acid (cDNA) into the cell’s genome, so that genetically modified cells become capable of expressing the functional ARSA enzyme. |
| List of Excipients |
|---|
|
Dimethylsulfoxide |
50 mL ethylene vinyl acetate (EVA) infusion bag(s) with two available spike ports, packed in an EVA overwrap bag placed inside a metal cassette.
Libmeldy is shipped from the manufacturing facility to the treatment centre storage facility in a cryoshipper, which may contain multiple metal cassettes intended for a single patient. Each metal cassette contains one infusion bag of Libmeldy.
Orchard Therapeutics (Netherlands) B.V., Basisweg 10, 1043 AP Amsterdam, The Netherlands
EU/1/20/1493/001
Date of first authorisation: 17 December 2020
| Drug | Countries | |
|---|---|---|
| LIBMELDY | Austria, Estonia, France, Croatia, Ireland, Italy, Lithuania |
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