Spain agrees to reimburse Orchard's gene therapy Libmeldy

Orchard Therapeutics has secured reimbursement from another European market for Libmeldy, an ex vivo gene therapy for the rare childhood disease metachromatic leukodystrophy (MLD) first approved in the EU in 2020.

The Interministerial Commission for the Pricing of Medicines in Spain has backed access by the Spanish National Health System (SNS) to Libmeldy (atidarsagene autotemcel) for all forms of early-onset MLD, including pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ).

The Spanish agreement follows similar reimbursement agreements in the UK, Germany, Italy, and other European countries and, as a result, Orchard is working to qualify its seventh European treatment centre for Libmeldy in Spain.

MLD is a previously untreatable genetic disorder that causes rapid deterioration in babies, leading to a loss of motor and cognitive function and eventual death. It's caused by a deficiency in the ARSA gene coding for the enzyme arylsulfatase-A, which allows sulphatide compounds to build up in the body.

Children with MLD eventually deteriorate into a vegetative state, which may require 24-hour intensive care, and the majority pass away within five years of symptom onset.

Libmeldy takes the form of an ex vivo gene therapy in which, following a round of chemotherapy, patients' blood stem cells are removed, genetically modified to include a functional copy of the ARSA gene, and then returned to the body, where they engraft within the bone marrow.

In Spain, it is assumed that two or three children born with MLD per year could potentially be eligible for the gene therapy, which has a list price of around €3 million per one-off treatment.

Orchard is now a unit of Japan's Kyowa Kirin, which bought the UK company in a deal valued at around $478 million that completed in January 2024. In calendar year 2024, Kowa Kirin recorded JPY 3.3 billion ($22 million) in Libmeldy sales, which was also approved in the US last year under the Lenmeldy brand name. Analysts have suggested that the gene therapy could achieve peak sales of around $300 million a year.

"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.