Medicine guide
Libmeldy
·
- Prescription status not stated
- Pain, sleep, mood, epilepsy and the brain
- Active substance
- Autologous CD34+ cells encoding ARSA gene
- Made by
- Orchard Therapeutics (Netherlands) B.V.
- Source
- Official label
Quoted from the official source. Not yet reviewed by Mediclarum — the leaflet in your pack is authoritative.
What it is for
- Libmeldy is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity: in children with late infantile or early juvenile forms, without clinical manifestations of the disease, in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.
From the official label · 2026-09-30 · European Medicines Agency
Details
| Made by | Orchard Therapeutics (Netherlands) B.V. |
|---|---|
| Active substance | Autologous CD34+ cells encoding ARSA gene |
| Used in | Pain, sleep, mood, epilepsy and the brain |
| Classification | N07 · Pain, sleep, mood, epilepsy and the brain |
| Orphan medicine | Yes — designated for a rare disease, according to the register |
| Additional monitoring ▼ | Yes — under additional monitoring (black triangle). Report suspected side effects. |
| Authorised on | 2020-12-17 |
| Last updated by the regulator | 2025-12-16 |
| EMA product number | EMEA/H/C/005321 |
Source: European Medicines Agency — medicine data · 2026-09-30 · not reviewed by a clinician
Source: European Medicines Agency (EMA). Product information as published by EMA. Reuse terms: EMA legal notice (reproduction authorised).