Libmeldy (Atidarsagene Autotemcel)

The active ingredient, atidarsagene autotemcel, consists of the patient’s own CD34+ hematopoietic stem cells that have been genetically modified with a lentiviral vector carrying a functional ARSA gene. After infusion, these cells engraft in the bone marrow and continuously produce arylsulfatase A, correcting the enzyme deficiency that underlies MLD.

The dosing regimen for Libmeldy is individualized. The prescribing physician determines the number of transduced stem cells to be infused based on the patient’s weight, disease severity, and manufacturing specifications. No fixed dose is publicly disclosed.

The safety of Libmeldy in pregnant or lactating individuals has not been established. Because the therapy involves genetically modified cells and a conditioning regimen, it is generally recommended to avoid use unless the potential benefit justifies the potential risk and a specialist is consulted.

To request Libmeldy, submit an enquiry on the product page at gnhindia.com with the required quantity. GNH India works with hospitals, pharmacies and procurement teams worldwide, verifies trade or institutional credentials, and then provides pricing, availability, cold‑chain shipping details and the necessary import documentation.

Libmeldy is a one‑time autologous gene‑therapy that aims to provide long‑term enzyme production, whereas conventional approaches such as hematopoietic stem‑cell transplantation rely on donor cells and may not achieve sustained enzyme levels. Enzyme‑replacement therapies are still investigational for MLD, making Libmeldy a distinct option with a different risk‑benefit profile.

Libmeldy must be stored refrigerated at 2‑8 °C. The product should be kept in its original container, protected from light, and must never be frozen or shaken. Temperature monitoring during transport is essential to maintain cell viability.

Libmeldy is delivered as an intravenous infusion of an autologous hematopoietic stem‑cell suspension. The infusion is performed in a specialized clinical setting after the patient has undergone a conditioning regimen to facilitate engraftment of the modified cells.

Serious risks include graft failure, insertional oncogenesis leading to malignancy, severe infections due to immunosuppression, and immune‑mediated reactions such as cytokine release syndrome. Continuous long‑term monitoring is required to detect and manage these potential complications.

Libmeldy is contraindicated in patients with active uncontrolled infections, those who cannot undergo the required myeloablative conditioning, or individuals with a history of malignancy related to prior gene‑therapy exposure. It is also not recommended for patients lacking a confirmed diagnosis of MLD with the appropriate disease onset.