Libmeldy is an ex vivo genetically modified autologous CD34+ haematopoietic stem and progenitor cell (HSPC) gene therapy for early onset MLD.1
Metachromatic leukodystrophy (MLD) is a rare, inherited, neurometabolic disease that causes rapid and progressive demyelination and neurodegeneration, leading to severe neurological disability and death.2 MLD is caused by mutations in the arylsulfatase A (ARSA) gene which lead to ARSA enzyme deficiency.1
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Libmeldy is indicated for the treatment of early onset MLD, in pre-symptomatic late infantile (LI) and early juvenile (EJ) and early-symptomatic EJ patients. CD34+ cells are transduced ex vivo with a lentiviral vector encoding ARSA cDNA. It is an autologous gene therapy, meaning a patient’s own stem cells are genetically corrected by inserting a functional copy of the ARSA gene and these are returned to their body.1
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Abbreviations
cDNA: complementary DNA.
▼ This medicinal product is subject to additional monitoring. Healthcare professionals are asked to report any suspected adverse events. Further information about local reporting details can be found in Section 4.8 of the Summary of Product Characteristics.
Please also report any adverse events to Orchard Therapeutics at: drugsafety@orchard-tx.com
For any medical questions, please contact medinfo@orchard-tx.com
References