Libmeldy (autologous CD34+ cells encoding ARSA gene)
- Region
- 🇪🇺 EU
- Active substance
- autologous CD34+ cells encoding ARSA gene
- Company
- Orchard Therapeutics (Netherlands) B.V.
- Status
- Authorised
- Therapeutic area
- Leukodystrophy, Metachromatic
- CHMP opinion
- Thu 15 Oct 2020
- EU approval
- Thu 17 Dec 2020
- Designations
- Orphan, ATMP, Additional monitoring
- Application
- EMEA/H/C/005321
- ATC
- N07
- Official record
- www.ema.europa.eu
Indication
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.
Regulatory milestones
No milestones recorded in the last year.
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.