Orchard Therapeutics, now under Kyowa Kirin, celebrates the FDA approval of Lenmeldy™ (atidarsagene autotemcel), previously OTL-200, for treating early-onset metachromatic leukodystrophy (MLD) in children. This milestone offers hope to affected families, as MLD typically results in rapid neurological decline and early mortality, with limited treatment options until now.
Lenmeldy addresses the genetic root of MLD by genetically modifying a patient’s own hematopoietic stem cells ex vivo, thereby inserting functional copies of the ARSA gene. Once reintroduced into the patient, these cells can potentially halt or slow disease progression by restoring enzyme function, crucial for breaking down fats causing neurological damage.
Lenmeldy™ (atidarsagene autotemcel), previously referred to as OTL-200, is an autologous hematopoietic stem cell-based gene therapy stands as the sole FDA-approved treatment in the United States designated for children grappling with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early-symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
Lenmeldy’s approval, following Priority Review and designations as a Rare Pediatric Disease and Regenerative Medicine Advanced Therapy, underscores its importance in addressing unmet medical needs. Additionally, Orchard Therapeutics’ receipt of a Priority Review Voucher, to be transferred to GSK per licensing agreements, further highlights the significance of this achievement.
Moving forward, there’s a collective call for universal newborn screening for MLD to ensure early diagnosis and timely intervention. This momentous approval marks a significant step towards improving outcomes for children with MLD, signaling a new era in the treatment of this devastating disease.