Lenmeldy FDA Approval History
Last updated by Judith Stewart, BPharm on March 18, 2024.
FDA Approved: Yes (First approved March 18, 2024)
Brand name: Lenmeldy
Generic name: atidarsagene autotemcel
Dosage form: Suspension for Intravenous Infusion
Previous Name: OTL-200
Company: Orchard Therapeutics
Treatment for: Metachromatic Leukodystrophy
Lenmeldy (atidarsagene autotemcel) is an autologous hematopoietic stem cell (HSC) gene therapy for the treatment of children with metachromatic leukodystrophy (MLD).
- Lenmeldy is indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
- Metachromatic leukodystrophy is a rare and life-threatening disease caused by a mutation in the arylsulfatase-A (ARSA) gene that results in reduction of ARSA enzymatic activity, leading to the accumulation of sulfatides in the brain and other areas of the body. Over time, damage to the nervous system leads to neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, and patients gradually lose the ability to move, talk, swallow, eat and see.
- Lenmeldy is an ex vivo genetically modified CD34+ autologous hematopoietic stem and progenitor cell (HSPC) gene therapy. Autologous CD34+ HSPCs are 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, which can break down, or prevent the build-up, of harmful sulfatides.
- Lenmeldy is a single-dose cell suspension administered by intravenous infusion. Mobilization, apheresis, and myeloablative conditioning are required prior to a Lenmeldy infusion.
- Warnings and precautions associated with Lenmeldy include thrombosis and thromboembolic events, encephalitis, serious infection, veno-occlusive disease, delayed platelet engraftment, risk of neutrophil engraftment failure, risk of insertional oncogenesis, and risk of hypersensitivity reactions.
- Common adverse reactions include febrile neutropenia (85%), stomatitis (77%), respiratory tract infections (54%), rash (33%), device related infections (31%), other viral infections (28%), pyrexia (21%), gastroenteritis (21%), and hepatomegaly (18%).
Common laboratory abnormalities include elevated D-dimer (67%), neutropenia (28%), and elevated liver enzymes (23%).
Development timeline for Lenmeldy
Further information
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