Posted by Michael Wonder on 22 Feb 2022
Health economic assessment of the gene therapy Libmeldy in metachromatic leukodystrophy
22 February 2022 - Within the FINOSE collaboration, TLV, together with the corresponding authorities in Finland and Norway, has produced a health economic assessment of the gene therapy Libmeldy (autologous CD34 positive, enriched cell fraction that contains the gene for aryl sulphatase A).
Clinical studies show that treatment with Libmeldy makes patients live longer and prolongs the time that patients retain their mobility. FINOSE assesses that the results are associated with uncertainties because few patients are included in the studies and the studies do not have placebo-treated controls. FINOSE assesses that there are uncertainties regarding the long-term effect and safety of the treatment.
