Registrational study of OTL-200 in patients with early-onset Metachromatic-leukodystrophy
Latest Information Update: 07 Feb 2025
Price :
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At a glance
- Drugs Atidarsagene autotemcel (Primary)
- Indications Metachromatic leukodystrophy
- Focus Registrational; Therapeutic Use
- Sponsors Orchard Therapeutics
- 03 Feb 2025 According to an Orchard Therapeutics media release, data from this trial will be presented at the 21st Annual WORLDSymposium taking place February 3-7 in San Diego.
- 20 Mar 2024 According to an Orchard Therapeutics media release, company announced the details of U.S. commercial launch of Lenmeldy (atidarsagene autotemcel) for the treatment of children early-onset metachromatic leukodystrophy (MLD). 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.
- 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.