Orchard Therapeutics has unveiled its groundbreaking treatment, Lenmeldy™ (atidarsagene autotemcel), as the first and only approved therapy designed for children afflicted with early-onset metachromatic leukodystrophy.
Lenmeldy™ (atidarsagene autotemcel), previously referred to as OTL-200, stands as the sole sanctioned treatment in the United States targeting children diagnosed with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early-symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
Lenmeldy represents a groundbreaking leap in medical science, offering the potential to halt or slow down the progression of this severe childhood condition with a single treatment, especially when administered before symptoms become apparent.
The core objective of Lenmeldy is to address the fundamental genetic origins of MLD by introducing one or more functional copies of the human ARSA gene ex vivo (outside the body) into the genome of a patient's haematopoietic stem cells (HSCs) using a lentiviral vector.
Once reintroduced into the patient's system, these genetically modified cells engraft and differentiate into various cell types, some of which traverse the blood-brain barrier into the central nervous system, where they express the requisite enzyme.
MLD is an extremely rare, rapidly progressive, and ultimately fatal neurometabolic disorder, affecting approximately one in 100,000 live births, translating to fewer than 40 children annually in the U.S.
This condition arises from a mutation in the gene encoding the enzyme arylsulfatase A (ARSA), leading to neurological deterioration and developmental regression. The majority of these children succumb within five years of symptom onset.
