Orchard Therapeutics Announces Reimbursement Agreement in Spain

Core Insights - Orchard Therapeutics has secured an agreement with the Spanish National Health System (SNS) for reimbursed access to Libmeldy® for eligible children with early-onset metachromatic leukodystrophy (MLD) [1][3] - MLD is an ultra-rare and fatal neurometabolic disease, with severe forms leading to rapid neurological decline and high mortality rates [2][4] - The agreement follows similar reimbursement arrangements in multiple European countries, enhancing treatment access for patients [3] Company Overview - Orchard Therapeutics, a Kyowa Kirin company, specializes in gene therapy aimed at treating severe genetic diseases through hematopoietic stem cell (HSC) gene therapy [8][9] - The company was founded in 2015 and has been pivotal in advancing HSC gene therapy from concept to clinical application [9] Product Information - Libmeldy® (atidarsagene autotemcel) is designed to correct the genetic defect causing MLD by inserting functional copies of the ARSA gene into the patient's own stem cells [5][6] - The treatment requires high-dose chemotherapy prior to the infusion of genetically modified cells, which can potentially halt or slow disease progression with a single administration [6] Market Context - MLD occurs in approximately one in every 100,000 live births, with an estimated two to three new cases annually in Spain, highlighting the rarity of the condition [4] - Early detection and diagnosis are critical for improving patient outcomes, prompting Orchard Therapeutics to support expanded newborn screening initiatives [4]