Orchard Therapeutics and Er-Kim Announce Partnership to Broaden Access to Libmeldy to Eligible Patients in Turkey and Certain Eurasian Countries

Core Insights - Orchard Therapeutics has entered into an exclusive distribution agreement with Er-Kim to commercialize Libmeldy® (atidarsagene autotemcel), the only approved therapy for children with early-onset metachromatic leukodystrophy (MLD) [1][3] Company Overview - Orchard Therapeutics, a Kyowa Kirin company, focuses on developing and commercializing gene therapies aimed at treating severe genetic diseases [10][11] - Er-Kim Pharmaceuticals, established in 1981, partners with over 40 global leaders and has a revenue exceeding EUR 200 million, serving over 600 million patients [9] Product Information - Libmeldy is designed to correct the genetic cause of MLD by inserting functional copies of the ARSA gene into a patient's hematopoietic stem cells [6][8] - The therapy requires patients to undergo high-dose chemotherapy prior to treatment to prepare for the infusion of genetically modified cells [7] Market Potential - MLD is an ultra-rare disease affecting approximately one in 100,000 live births, with a higher incidence in Turkish and Eurasian populations [2][5] - The partnership aims to enhance access to Libmeldy in Turkey, Russia, Ukraine, and the Commonwealth of Independent States, addressing a significant unmet medical need in these regions [3][4]