Pharma Focus Europe

Orchard Therapeutics Enters Agreement with Beneluxa Consortium to Facilitate Reimbursed Access to Libmeldy

Friday, January 26, 2024

Orchard Therapeutics, a prominent global gene therapy company, has recently announced a new agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa). This agreement aims to facilitate reimbursed access to Libmeldy® (atidarsagene autotemcel), a gene therapy utilizing hematopoietic stem cells (HSCs) for the treatment of early-onset metachromatic leukodystrophy (MLD). The agreement covers several member countries, including Belgium, The Netherlands, and Ireland. Similar agreements have been previously established with reimbursement authorities in the United Kingdom, Italy, Germany, Sweden, Iceland, Finland, and Norway, aligning with Orchard Therapeutics' strategic expansion plans.

MLD is a rare and fatal genetic disorder caused by a mutation in the gene responsible for encoding the enzyme arylsulfatase A (ARSA). This mutation leads to severe neurological damage and developmental regression, especially in infants. Libmeldy addresses this genetic anomaly by introducing a functional copy of the ARSA gene into the patient's own HSCs. The genetically repaired cells are then reintroduced into the patient, allowing them to naturally migrate into the central nervous system, engraft, and express the functional enzyme. This innovative approach holds promise for persistently restoring enzymatic function with a single treatment.

Clinical trials have demonstrated that Libmeldy can preserve cognitive development and maintain motor function beyond the ages at which untreated patients typically experience severe impairments. With over 250 cumulative patient-years of follow-up, Libmeldy has shown a favorable safety profile, with no treatment-related serious adverse events or deaths. The therapy has received approval from the European Commission (EC) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). In the United States, where it is known as OTL-200, Libmeldy is currently under Priority Review by the Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) goal date set for March 18, 2024.

Robin Kenselaar, Senior Vice President, EMEA at Orchard Therapeutics, expressed satisfaction with the agreement, highlighting the continued recognition of Libmeldy's impact by health technology assessment authorities across Europe. The agreement with Beneluxa followed the acceptance of additional long-term data and marks a positive outcome after earlier negotiations without reaching an arrangement. Orchard Therapeutics remains committed to expanding access to this critical therapy in new geographic regions for eligible patients and their families.

In addition to this agreement, Orchard Therapeutics provided updates on multiple eligible MLD patients currently in the treatment process, with the first patient treated at the qualified center in Utrecht, The Netherlands. The company's qualified treatment centers in Europe are expected to administer Libmeldy to additional patients in the coming months. Furthermore, centers in Spain and Saudi Arabia are in the final stages of qualification to treat MLD patients with Libmeldy in the near future.

Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease affecting the body's metabolic system. Libmeldy, developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, has been approved for eligible patients in the European Union, UK, Iceland, Switzerland, Liechtenstein, and Norway. It is also under investigation in the United States as OTL-200.



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