Trial Profile
A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)
Status:
Active, no longer recruiting
Phase of Trial:
Phase II
Latest Information Update: 21 Mar 2024
Price :
$35
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At a glance
- Drugs Atidarsagene autotemcel (Primary)
- Indications Metachromatic leukodystrophy
- Focus Registrational; Therapeutic Use
- Sponsors GlaxoSmithKline; Orchard Therapeutics
- 18 Mar 2024 According to an Orchard Therapeutics media release, the FDA approval of Lenmeldy is based on data from 37 pediatric patients with early-onset MLD, enrolled in two single-arm, open-label clinical studies or treated under European expanded access frameworks, who received a one-time administration of the gene therapy and compared with natural history data.
- 18 Mar 2024 According to an Orchard Therapeutics media release, company announced the U.S. Food and Drug Administration (FDA) has approved Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) collectively referred to as early-onset metachromatic leukodystrophy (MLD).
- 13 Nov 2023 According to an Orchard Therapeutics media release, in September, the U.S. Food and Drug Administration (FDA) accepted the filing of the Biologics License Application (BLA) for OTL-200 in metachromatic leukodystrophy (MLD) under Priority Review.