– First and only disease-modifying therapy for the treatment of eligible children with MLD also receives Orphan Drug Designation and Priority Review in the Kingdom of Saudi Arabia –
TOKYO, LONDON and BOSTON, Oct. 28, 2025 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd., (TSE: 4151, President and COO: Abdul Mullick, "Kyowa Kirin") and Orchard Therapeutics announced that Japan's Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Regenerative Medicine Product Designation to OTL-200, also known as atidarsagene autotemcel, an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), and early-symptomatic early juvenile (ESEJ)—collectively referred to as early-onset—metachromatic leukodystrophy (MLD).
MLD is an ultra-rare, rapidly progressive, irreversible and ultimately fatal neurometabolic disease that affects approximately one in 100,000 live births based on existing literature. It is caused by an error in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neurological damage and developmental regression. In the most severe form of MLD, babies develop normally but in late infancy start to rapidly lose the ability to walk, talk and interact with the world around them. These children eventually deteriorate into a vegetative state, which may require 24-hour intensive care, and the majority pass away within five years of symptom onset, creating an enormous emotional and financial burden on the family. There are currently no approved therapies for MLD in Japan beyond supportive and end-of-life care.
OTL-200 aims to correct the underlying genetic cause of MLD by inserting one or more functional copies of the human ARSA gene ex vivo (outside the body) into the genome of a patient’s own hematopoietic stem cells (HSCs) using a lentiviral vector. The genetically repaired cells are infused back into the patient, where, once engrafted, they differentiate into multiple cell types, some of which migrate across the blood-brain barrier into the central nervous system and express the functional enzyme. Kyowa Kirin is currently preparing the potential initiation of a clinical trial for OTL-200 in children with PSLI, PSEJ, and ESEJ MLD in Japan.
In addition, OTL-200 has received Orphan Drug Designation and Priority Review from the Saudi Food and Drug Authority (SFDA) for the treatment of early-onset MLD. The SFDA Orphan Drug Designation and Priority Review program are intended to encourage development of new therapies for rare and ultra-rare diseases by streamlining and expediting the review process. Concurrently, Orchard Therapeutics is working to qualify a treatment center in the Kingdom of Saudi Arabia to administer the therapy to eligible children with MLD from the country and surrounding regions through pre-approval access pathways and eventually commercially, if approved.
“We are very pleased that OTL-200 has received designation as an Orphan Regenerative Medicine Product in Japan,” said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “This designation underscores the importance of OTL-200 as an innovative treatment for young patients suffering from MLD, a life-threatening and severe neurodegenerative disease for which no treatment has existed until now. OTL-200 has also received a similar designation in Saudi Arabia. This therapy is already being used in Europe and the United States, and it is highly regarded as a treatment that can restore enzymatic function with a single administration and has the potential to slow the progression of this severe neurodegenerative disease. We are committed to promptly advancing the development of OTL-200 to deliver life-changing value to patients and their families in Japan, working to make this therapy available to patients in other regions worldwide.”
OTL-200, approved as Lenmeldy™ in the United States (U.S.) and Libmeldy® in Europe, is the only therapy intended to correct the underlying cause of MLD for eligible patients.
“Our hematopoietic stem cell gene therapy approach continues to demonstrate great promise in addressing diseases for which current treatments are limited or do not exist,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. “Bringing these potentially transformative therapies to children and families in need is central to our mission of ending the devastation caused by severe genetic diseases, and these designations represent important progress toward advancing OTL-200 toward a potential regulatory approval in Japan and beyond.”
The Kyowa Kirin Group companies strive to contribute to the health and well-being of people around the world by creating new value through the pursuit of advances in life sciences and technologies.
About MLD
MLD is a rare and life-threatening inherited disease of the body’s metabolic system estimated to occur in approximately one in every 100,000 live births. MLD is caused by an error in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged, leading to neurological problems such as motor, behavioral and cognitive regression, severe spasticity, and seizures. Patients with MLD gradually lose the ability to move, talk, swallow, eat and see. In its late infantile form, mortality at five or ten years from onset is estimated at 75 percent and 100 percent, respectively.i
About Kyowa Kirin
Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin’s values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.
About Orchard Therapeutics
Orchard Therapeutics, a Kyowa Kirin company, is a global gene therapy leader focused on ending the devastation caused by genetic and other severe diseases by discovering, developing, and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patient’s own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease with a single treatment.
Founded in 2015, Orchard’s roots go back to some of the first research and clinical developments involving HSC gene therapy. Our team has played a central role in the evolution of this technology from a promising scientific idea to a potentially life-transforming reality. Today, as a vital part of Kyowa Kirin’s global business, Orchard is continuing to advance a pipeline of HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.
For more information, please visit www.orchard-tx.com.
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iMahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669
CONTACT: CONTACT For Kyowa Kirin Kyowa Kirin (Tokyo, Japan) Hiroki Nakamura, +81-3-5205-7205 media@kyowakirin.com For Orchard Therapeutics Benjamin Navon +1 857-248-9454 Benjamin.Navon@orchard-tx.com
