Orchard Therapeutics Announces Reimbursement Agreement in Spain

LONDON, MADRID, and BOSTON, Feb. 28, 2025 (GLOBE NEWSWIRE) —  Orchard Therapeutics, a Kyowa Kirin company, announced it has recently reached an agreement with the Interministerial Commission for the Pricing of Medicines of the Directorate of Common Portfolio of Services of the Spanish National Health System (SNS) which will result in reimbursed access to Libmeldy® (atidarsagene autotemcel) for all eligible children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or 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 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.

“Libmeldy opens up tremendous new possibilities for children in Spain with early-onset MLD who previously had no treatment options beyond supportive and end-of-life care,” said Cristiana Giani, country manager of Italy and Iberia at Orchard Therapeutics. “We are grateful for the collaboration with the SNS to come to an agreement that enables appropriately broad and sustainable access to treatment for eligible patients and their families.”

The agreement with SNS follows similar arrangements made with reimbursement authorities in the United Kingdom (UK), Ireland, Germany, Italy, Sweden, Iceland, Finland, Norway, Belgium and The Netherlands. With reimbursed access now secured, the company is working to qualify its seventh European treatment center in Spain.

MLD is estimated to occur in approximately one in every 100,000 live births based on existing literature. In Spain, it is assumed that two or three children born with MLD could be potentially eligible for the treatment annually. As with many rare, life-threatening pediatric diseases, early detection and diagnosis is key to ensuring the best possible outcomes for patients, which is why Orchard Therapeutics supports efforts to expand newborn screening for diseases like MLD which meet widely accepted inclusion criteria for population-based screening.

About Libmeldy
Libmeldy^® (atidarsagene autotemcel) aims to correct the underlying genetic cause of metachromatic leukodystrophy (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.

Prior to treatment, patients must undergo high-dose chemotherapy, a process that removes cells from the bone marrow so they can be replaced with the modified cells in Libmeldy. This approach has the potential to restore enzymatic function to stop or slow disease progression with a single treatment.

Libmeldy is approved by the European Commission (EC) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). For more information, please see the Summary of Product Characteristics (SmPC) available on the EMA website.

The program was originated by and developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy.

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, Orchard is advancing 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 https://orchard-tx.eu.

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, we have invested in drug discovery and biotechnology innovation for more than 70 years and are 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 our values, to sustainable growth, and to making people smile unites us across the globe. You can learn more about the business of Kyowa Kirin at www.kyowakirin.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

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