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Registrational study of OTL-200 in patients with early-onset Metachromatic-leukodystrophy

Trial Profile

Registrational study of OTL-200 in patients with early-onset Metachromatic-leukodystrophy

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 21 Mar 2024

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At a glance

  • Drugs Atidarsagene autotemcel (Primary)
  • Indications Metachromatic leukodystrophy
  • Focus Registrational; Therapeutic Use
  • Sponsors Orchard Therapeutics
  • Most Recent Events

    • 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 The U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel) to Orchard Therapeutics, 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).
    • 02 Feb 2024 According to an Orchard Therapeutics media release, long-term results from an updated integrated analysis of 39 patients with MLD treated with investigational OTL-200 from this trial will be featured at the 20th Annual WORLDSymposium™ taking place February 4-9, 2024, in San Diego, California.
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