Atidarsagene autotemcel
- Generic Name
- Atidarsagene autotemcel
- Brand Names
- Libmeldy
- Drug Type
- Biotech
- Unique Ingredient Identifier
- EPP8G99QG4
Overview
Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy comprising genetically modified autologous CD34+ enriched hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic leukodystrophy (MLD).
Background
Atidarsagene autotemcel, sold under the brand name Libmeldy, is a gene therapy comprising genetically modified autologous CD34+ enriched hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic leukodystrophy (MLD).
Indication
Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity in children with:
Associated Conditions
- Metachromatic Leukodystrophy (MLD)
Clinical Trials
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No clinical trials found
No clinical trials found for this drug
FDA Approved Products
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No FDA products found
No FDA products found for this drug
EMA Approved Products
Medicine Name | EMA Number | Auth. Holder | Country | Drug Type | Status | Issued | Opinion | Revision |
|---|---|---|---|---|---|---|---|---|
Libmeldy | EMEA/H/C/005321 | Orchard Therapeutics (Netherlands) BV,Prins Bernhardplein 200,1097 JB Amsterdam,The Netherlands | the netherlands | Orphan | Authorised | 2020/12/17 | 2020/10/15 | 6 |
Singapore Approved Products
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No Singapore products found
No Singapore products found for this drug
China Approved Products
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No China products found
No China products found for this drug
Australia Approved Products
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No Australia products found
No Australia products found for this drug