Long-term clinical outcomes with Libmeldy in children with early-onset MLD

Gene therapy developer Orchard Therapeutics (Nasdaq: ORTX) has announced the publication in The Lancet of long-term clinical outcomes evaluating the safety and efficacy of Libmeldy (atidarsagene autotemcel) for the treatment of early-onset metachromatic leukodystrophy (MLD).

Libmeldy is the only approved one-time gene therapy intended to correct the underlying cause of MLD for eligible patients in the European Union, UK, Iceland, Liechtenstein and Norway. Also known as OTL-200, it is an investigational therapy in the USA.

“MLD is a cruel and ultimately fatal disease for which there were previously no approved treatment options beyond supportive care,” said Professor Alessandro Aiuti, deputy director of the San Raffaele Telethon Institute for Gene Therapy in Milan and full professor of pediatrics at the Vita-Salute San Raffaele University of Milan and a senior author of The Lancet manuscript. “Libmeldy represents a significant step forward in the treatment of MLD. These data highlight the potential long-term benefits of HSC gene therapy for these children, especially when intervention prior to symptom onset is possible,” he noted.