Orchard Therapeutics has reached a significant agreement with the Interministerial Commission on Drug Pricing in Spain, which will allow the inclusion of its gene therapy Libmeldy® in the pharmaceutical provision of the National Health System (NHS). This advancement will benefit children suffering from early-onset metachromatic leukodystrophy, a rare and devastating neurometabolic disease that affects neurological development in childhood.
Metachromatic leukodystrophy is characterized by a progressive deterioration that, if left untreated, can lead to a vegetative state and death within five years of symptom onset. This disease is caused by a failure in the gene that encodes the enzyme arylsulfatase A, crucial for the neurological health of children.
Cristiana Giani, country manager for Italy and Iberia at Orchard Therapeutics, expressed satisfaction with the agreement reached, highlighting that “Libmeldy opens new and enormous possibilities for children in Spain with early-onset metachromatic leukodystrophy, who previously had no treatment options beyond supportive care.” This treatment offers new hope, especially considering that affected children generally lack effective therapeutic alternatives.
The agreement adds to others signed in countries such as the UK, Ireland, and Germany, emphasizing the importance of early diagnosis. It is estimated that annually, between two and three children in Spain could be eligible for this treatment. Orchard Therapeutics is also committed to supporting newborn screening for the disease to ensure its detection as early as possible.
Libmeldy® (atidarsagene autotemcel) acts by correcting the genetic cause of leukodystrophy through an innovative technique that uses the patient’s own hematopoietic stem cells. After receiving treatment with chemotherapy that prepares the patient for the introduction of genetically modified cells, the goal is to restore enzymatic function and slow or even stop the progression of the disease with a single intervention.
The inclusion of Libmeldy® in the NHS not only represents a medical advancement, but also improves the quality of life for children and their families, who have faced the heavy burden of this serious condition. Orchard Therapeutics, in collaboration with Kyowa Kirin, continues to develop therapeutic solutions for rare diseases, marking an important step in the fight against genetic disorders.
Source: MiMub in Spanish
