Orchard Therapeutics and Er-Kim Partner to Expand Libmeldy Access in Turkey and Eurasia

Orchard Therapeutics, recently acquired by Kyowa Kirin, has entered into an exclusive distribution agreement with Er-Kim, a global pharmaceutical company specializing in new therapies. This partnership aims to commercialize Libmeldy® (atidarsagene autotemcel), the only approved treatment for children with early-onset metachromatic leukodystrophy (MLD). Libmeldy addresses the genetic cause of MLD, a rare and rapidly progressing neurometabolic disease affecting approximately one in 100,000 live births.

Under this agreement, Er-Kim will act as Orchard Therapeutics' exclusive representative to identify and refer eligible children for Libmeldy treatment across Turkey, Russia, Ukraine, and the Commonwealth of Independent States. These children will be treated at qualified centers in Europe. Cem Zorlular, CEO of Er-Kim, expressed excitement about the partnership, highlighting the transformative potential of Libmeldy for children with MLD who previously had no approved treatment options beyond supportive and palliative care.

MLD is a severe inherited metabolic disorder caused by mutations in the ARSA gene, leading to the accumulation of harmful substances in the brain and other organs, including the liver, gallbladder, and kidneys. This accumulation damages the nervous system, resulting in severe neurological symptoms such as motor and cognitive regression, spasticity, and seizures. Over time, patients lose their ability to move, speak, swallow, and see. The disease is life-threatening, with a high mortality rate within five years of onset for late infantile forms and ten years for juvenile forms.

Robin Kenselaar, Senior Vice President and General Manager of EMEA at Orchard Therapeutics, noted that MLD has a higher incidence in Turkish and Eurasian populations compared to the U.S. and Europe. This partnership with Er-Kim aims to expand access to Libmeldy in these regions, leveraging Er-Kim's extensive regional expertise in delivering specialty medicines.

Libmeldy works by inserting functional copies of the ARSA gene into a patient's hematopoietic stem cells (HSCs) using a lentiviral vector. These genetically modified cells are then infused back into the patient after high-dose chemotherapy, which clears the bone marrow to accommodate the new cells. Once engrafted, these cells differentiate into various cell types, some of which migrate to the central nervous system and produce the necessary enzyme to combat MLD. This treatment has the potential to restore enzymatic function and halt or slow disease progression with just one treatment.

Libmeldy is approved by multiple regulatory agencies, including the European Commission, UK Medicines and Healthcare products Regulatory Agency, and Swissmedic. The therapy was developed in collaboration with the San Raffaele-Telethon Institute for Gene Therapy in Italy.

Er-Kim Pharmaceuticals, founded in 1981, is recognized for its innovative approach in biopharmaceuticals, collaborating with over 40 global leaders to enhance patient care in key markets. With over 280 dedicated professionals and revenues surpassing EUR 200 million, Er-Kim's commitment to healthcare innovation continues to set new standards in patient access and commercialization.

Orchard Therapeutics, a leader in global gene therapy, is committed to transforming the lives of patients with genetic and severe diseases through hematopoietic stem cell gene therapy. This approach involves modifying a patient's own blood stem cells to treat the underlying disease cause, offering a potentially curative treatment with a single administration.

Kyowa Kirin, a Japan-based global specialty pharmaceutical company, focuses on discovering and delivering breakthrough treatments. With over 70 years of innovation in drug discovery and biotechnology, Kyowa Kirin works on developing advanced therapies for high unmet medical needs, including rare diseases and intractable hematological conditions.