New FDA Approval: LENMELDY™ (atidarsagene autotemcel) Summary
The Food and Drug Administration (FDA) approved LENMELDY™ (atidarsagene autotemcel), the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).
| Drug Name |
LENMELDY™ (atidarsagene autotemcel) |
| Active Ingredient |
atidarsagene autotemcel |
| Date of Approval |
3/18/2024 |
| Manufacturer |
Orchard Therapeutics |
| Approval Pathways |
BLA |
| Therapeutic Class |
Gene Therapy |
| Formulation |
Suspension for Intravenous Infusion |
| MoA |
LENMELDY™ inserts one or more functional copies of the human ARSA complementary deoxyribonucleic acid (cDNA) into the patients’ HSCs, through transduction of autologous CD34+ cells with ARSA LVV.
After LENMELDY™ infusion, transduced CD34+ HSCs engraft in bone marrow, repopulate the hematopoietic compartment and their progeny produce ARSA enzyme. Functional ARSA enzyme can breakdown or prevent the harmful accumulation of sulfatides.
|
| Dosing and Administration |
LENMELDY™ is a single-dose cell suspension for intravenous infusion.
LENMELDY™ is composed of one to eight infusion bags which contain 2 to 11.8× 106 cells/mL (1.8 to 11.8 x 106 CD34+ cells/ml) suspended in cryopreservation solution.
For autologous use only. For one-time single-dose intravenous use only.
• Children are required to undergo hematopoietic stem cell (HSC) mobilization followed by apheresis to obtain CD34+ cells for LENMELDY manufacturing.
• Dosing of LENMELDY™ is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight.
• The minimum recommended dose is based on the MLD disease subtype.
• Myeloablative conditioning must be administered before infusion of LENMELDY™.
• Confirm that the child’s identity matches the unique patient identification information on the LENMELDY™ infusion bag(s) prior to infusion.
• Do not sample, alter, irradiate, or refreeze LENMELDY™.
• Do not use a leukodepleting filter.
|
| Place in Therapy |
This is the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Prior to treatment, patients must undergo high-dose chemotherapy, a process that removes cells from the bone marrow so they can be replaced with the modified cells in LENMELDY™. |
| Expected Market Launch Date |
Unavailable |
| New Molecular Entity (NME) or Existing Formulation |
NME |
| Expected Cost |
Unavailable |
| Product Discontinuation |
N/A |
Share