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Prescribing Information SmPC

LIBMELDY clinical study design

In an integrated data set, 29 children with MLD (16 late infantile, 13 early juvenile) aged 7.6‒139.9 months were treated with Libmeldy as part of the safety analysis and 25 children with MLD were included in the efficacy analysis. They were compared with 31 untreated children with MLD (19 late infantile, 12 early juvenile). The median duration of follow-up was 3.16 years (range 0.64-7.51 years). The aim of the study was to determine the efficacy and safety of Libmeldy hematopoietic stem cell (HSC) gene therapy. Of the 29 patents included (who were given the fresh formulation (OTL0200-f), 20 were from Study 201222 (Registrational study), 1 was from CUP 207394 (Compassionate Use Programme), 3 were from HE 205029 (Hospital exemption) and 5 were from SUP 206258 (Compassionate Use Programme).1

Summary of demographic characteristics by symptomatic status at time of gene therapy and by disease subtype (Integrated efficacy set)

Libmeldy Summary of Product Characteristics.2

Primary endpoints

The co-primary endpoints were, at 2 years post-treatment:3,4

  • An improvement of 10% of the total GMFM-88 score in treated patients compared to natural history.
  • A significant (≥2 SD) increase of ARSA activity from pre-treatment values in PBMCs.

At year 3, an interim safety and efficacy analysis was planned and, at year 8, an efficacy and safety follow-up was planned.

GMFM, gross motor function measurement; PBMC, peripheral blood mononuclear cells


  1. Fumagalli F, et al. Lentiviral hematopoietic stem cell gene therapy (HSC-GT) for metachromatic leukodystrophy (MLD) provides sustained clinical benefit; Presented at: 2019 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM); September 3-6, 2019; Rotterdam, The Netherlands.
  2. Libmeldy, Summary of Product Characteristics.
  3. Gene Therapy for Metachromatic Leukodystrophy (MLD). Available at: Accessed December 2020.
  4. Sessa M, et al. Lancet 2016; 388(10043): 476-487.