Orchard Therapeutics announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD). Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients? own HSCs.

The genetically modified cells are infused back into the patient, where they can naturally migrate across the blood-brain barrier into the central nervous system, engraft, and express the functional enzyme. This approach has the potential to persistently restore enzymatic function with a single treatment. In clinical trials, treatment with Libmeldy resulted in the preservation of cognitive development and maintenance of motor function past ages at which untreated patients showed severe cognitive and motor impairments.

With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths. Most adverse events were associated with busulfan conditioning or background disease. Libmeldy was previously approved by the European Commission (EC) and UK Medicines and Healthcare products Regulatory Agency (MHRA).

Libmeldy is known as OTL-200 in the U.S., where it is currently under Priority Review by the Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) goal date of March 18, 2024.