Spain approves reimbursement for Orchard’s gene therapy Libmeldy

SPAIN – Spain has approved reimbursement for Libmeldy, a gene therapy developed by Orchard Therapeutics to treat metachromatic leukodystrophy (MLD), a rare and fatal genetic disorder in children.

The decision, made by the Interministerial Commission for the Pricing of Medicines, allows the Spanish National Health System (SNS) to cover the therapy for all forms of early-onset MLD, including:

• Pre-symptomatic late infantile (PSLI)
• Pre-symptomatic early juvenile (PSEJ)
• Early symptomatic early juvenile (ESEJ)

This approval follows similar reimbursement agreements in Germany, Italy, the UK, and other European nations.

In response, Orchard is preparing its seventh European treatment center in Spain to support the therapy’s rollout.

A breakthrough for a previously untreatable disease

MLD is a devastating disorder caused by a deficiency in the ARSA gene, leading to the accumulation of harmful sulfatides in the body.

This results in rapid neurological decline, loss of motor and cognitive abilities, and eventual death.

Children with MLD typically do not survive beyond five years after symptoms appear and often require 24-hour intensive care in their final stages.

Libmeldy is an ex vivo gene therapy, meaning the treatment occurs outside the body. The process involves:

1. Extracting the patient’s blood stem cells
2. Genetically modifying them to include a working copy of the ARSA gene
3. Reinfusing the corrected cells into the patient after chemotherapy

Once inside the body, the modified cells integrate into the bone marrow and begin producing the missing arylsulfatase-A enzyme, halting disease progression.

Spain’s estimated patient numbers and cost

Spain estimates that two to three children are born with MLD each year and could qualify for Libmeldy.

The one-time therapy carries a list price of approximately €3 million (US $3.13 million) per patient. While expensive, the treatment offers the potential for long-term survival and a dramatically improved quality of life, replacing the need for palliative care.

Libmeldy’s commercial outlook

Orchard Therapeutics is now part of Kyowa Kirin, a Japanese pharmaceutical company that acquired it for US $478 million in January 2024.

In the same year, Kyowa Kirin reported JPY 3.3 billion (US $22 million) in Libmeldy sales. Analysts predict annual peak sales could reach US $300 million globally.

Libmeldy also received approval in the United States, where it is marketed under the name Lenmeldy.

Cristiana Giani, Orchard’s country manager for Italy and Iberia, described the approval as a game-changer for Spanish children with early-onset MLD.

“Libmeldy opens up tremendous new possibilities for children in Spain who previously had no treatment options beyond supportive and end-of-life care,” she said.