Registrational study of OTL-200 in patients with early-onset Metachromatic-leukodystrophy
Latest Information Update: 21 Mar 2024
At a glance
- Drugs Atidarsagene autotemcel (Primary)
- Indications Metachromatic leukodystrophy
- Focus Registrational; Therapeutic Use
- Sponsors 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 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.