LIBMELDY Dispersion for infusion Ref.[27971] Active ingredients: Atidarsagene autotemcel

Source: European Medicines Agency (EU)  Revision Year: 2023  Publisher: Orchard Therapeutics (Netherlands) B.V., Basisweg 10, 1043 AP Amsterdam, The Netherlands

Product name and form

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.

Qualitative and quantitative composition

2.1 General description

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.

2.2 Qualitative and quantitative composition

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
Sodium chloride
Human albumin

Pack sizes and marketing

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.

Marketing authorization holder

Orchard Therapeutics (Netherlands) B.V., Basisweg 10, 1043 AP Amsterdam, The Netherlands

Marketing authorization dates and numbers

EU/1/20/1493/001

Date of first authorisation: 17 December 2020

Drugs

Drug Countries
LIBMELDY Austria, Estonia, France, Ireland, Italy, Lithuania

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