The Swiss Agency for Therapeutic Products has granted approval for Orchard Therapeutics’ hematopoietic stem cell (HSC) gene therapy, Libmeldy (atidarsagene autotemcel), to treat early-onset metachromatic leukodystrophy (MLD).
Libmeldy introduces a functional copy of the arylsulfatase A (ARSA) gene into the genome of patients’ HSCs to rectify the underlying genetic cause of MLD.
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The genetically edited cells are delivered back into the patient, where they move across the blood-brain barrier into the central nervous system. The cells then engraft and express the functional enzyme.
This method could aid in restoring enzymatic activity through a single treatment.
Treatment with Libmeldy in clinical trials was shown to preserve cognitive development and uphold motor function from points at which untreated individuals had serious motor and cognitive impairments.
The therapy was found to be well-tolerated without treatment-associated serious adverse events or mortalities.
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By GlobalDataMost adverse events reported were linked to busulfan conditioning or background ailments.
Orchard Therapeutics chief medical officer Leslie Meltzer stated: “The approval of Libmeldy in Switzerland opens up tremendous new possibilities for eligible children with MLD who previously had no approved treatment options beyond supportive care.
“We are encouraged that regulatory authorities in the European region continue to recognise the clinical impact of Libmeldy, and we remain committed to broadening access to this important therapy in new geographies for the benefit of patients and their families.”
Developed in collaboration with the San Raffaele-Telethon Institute for Gene Therapy, Libmeldy is approved in the European Union, Iceland, Liechtenstein, Norway, Switzerland and the UK to treat MLD caused by biallelic mutations in the ARSA gene.
Orchard is progressing a pipeline comprising pre-clinical, clinical and commercial-stage HSC gene therapies.
The company acquired a rare disease gene therapy portfolio from GSK in 2018.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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