Orchard Therapeutics Announces Agreement with Spanish Health System for Reimbursed Access to Libmeldy® for Early-Onset Metachromatic Leukodystrophy Patients
London, Madrid, and Boston, Feb. 28, 2025 – Orchard Therapeutics, a Kyowa Kirin company, recently made an important announcement regarding the availability of Libmeldy® (atidarsagene autotemcel) for eligible children with early-onset metachromatic leukodystrophy (MLD) in the Spanish National Health System (SNS).
What is Metachromatic Leukodystrophy (MLD)?
Metachromatic leukodystrophy is a rare, progressive, and debilitating genetic disorder that affects the central and peripheral nervous system. It is caused by a deficiency in the enzyme arylsulfatase A, which is responsible for breaking down a specific fatty substance called sulfatides. The accumulation of these sulfatides leads to the demyelination of nerve fibers, causing symptoms such as developmental delays, motor impairment, and cognitive decline.
The Agreement and Its Impact on Patients
The agreement reached between Orchard Therapeutics and the Interministerial Commission for the Pricing of Medicines of the Directorate of Common Portfolio of Services of the Spanish National Health System will result in reimbursed access to Libmeldy® for all eligible children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ) MLD. This is a significant milestone for these patients, as it provides them with access to a potentially life-changing treatment at no cost to their families.
Understanding Libmeldy® and Its Benefits
Libmeldy® is a gene therapy designed to address the root cause of MLD by introducing a functional copy of the arylsulfatase A gene into the patient’s own hematopoietic stem and progenitor cells (HSPCs). These cells are then infused back into the patient’s body, where they begin to produce the missing enzyme. By doing so, Libmeldy® aims to halt the progression of the disease and improve the quality of life for patients.
The Global Impact of the Agreement
This agreement between Orchard Therapeutics and the Spanish National Health System marks a critical step forward in the treatment of MLD and sets a positive precedent for other healthcare systems around the world. It is expected that this agreement will lead to increased access to Libmeldy® for eligible patients in other regions, ultimately improving the lives of countless individuals and their families.
Conclusion
The recent agreement between Orchard Therapeutics and the Spanish National Health System is a significant advancement in the treatment of early-onset metachromatic leukodystrophy. This gene therapy, which introduces a functional copy of the arylsulfatase A gene into a patient’s own HSPCs, has the potential to halt the progression of the disease and improve the quality of life for eligible patients. The agreement’s global impact is expected to lead to increased access to Libmeldy® for eligible patients in other regions, providing hope to countless families affected by this debilitating condition.
- Orchard Therapeutics announces agreement with Spanish National Health System for reimbursed access to Libmeldy® for early-onset MLD patients.
- Libmeldy® is a gene therapy designed to address the root cause of MLD by introducing a functional copy of the arylsulfatase A gene into a patient’s own HSPCs.
- This agreement sets a positive precedent for other healthcare systems around the world, ultimately improving the lives of countless individuals and their families.
