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

More general terms

• gene therapy