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atidarsagene autotemcel (Lenmeldy)
Indications:
- autologous hematopoietic stem cell-based gene therapy indicated for treatment of children with metachromatic leukodystrophy
- pre-symptomatic late infantile
- pre-symptomatic early juvenile
- early symptomatic
- early juvenile
Contraindications:
- none
Dosage:
- single-dose cell suspension for intravenous infusion.
- 1-8 infusion bags which contain 2 to 11.8 x 10E6 cells/mL
Adverse effects:
- most common non-laboratory adverse reaction (>= 10%)
- febrile neutropenia (85%)
- stomatitis (77%)
- respiratory tract infections (54%)
- rash (33%)
- device related infections (31%)
- other viral infections (28%)
- pyrexia (21%)
- gastroenteritis (21%)
- hepatomegaly (18%)
- most common laboratory abnormalities
- elevated D-dimer (67%)
- neutropenia (28%)
- elevated serum transaminases (23%)
- warnings & cautions
- thrombosis & thromboembolism
- encephalitis
- serious infectiom
- veno-occlusive disease
- delayed platelet engraftment
- failure of neutrophil engraftment
- risk of insertional oncogenesis
- hypersensitivity
Drug interactions:
- anti-retroviral agents
- do not take anti-retroviral medications for at least one month prior to initiating medications for stem cell mobilization & for the duration of time needed for elimination of the medications
Mechanism of action:
- autologous hematopoietic stem cell-based gene therapy
General
gene therapy
References
- HIGHLIGHTS OF PRESCRIBING INFORMATION
LENMELDY (atidarsagene autotemcel) suspension for intravenous infusion
https://www.fda.gov/media/177109/download