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Prescribing Information SmPC

Indication

LibmeldyTM is indicated for the treatment of metachromatic leukodystrophy (MLD) characterised by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:1

– In children with late infantile or early juvenile forms, without clinical manifestations of the disease,

– In children with the early juvenile form, with the ability to walk independently and before the onset of cognitive decline*

*Early symptomatic: treatment with Libmeldy of a patient with an early-symptomatic early juvenile form of the disease should be considered:1

– If this patient is able to walk independently, which means that the patient’s GMFC-MLD score is ≤1, and

– If the patient’s cognitive function has not started declining, which means that the patient’s IQ is ≥85.

Abbreviations

ARSA: arylsulfatase A; EJ: early juvenile; GMFC: gross motor function classification; HSC: haematopoietic stem cell; IQ: intelligence quotient; MLD: metachromatic leukodystrophy; 0/0: null/null; 0/R: null/residual; R/R: residual/residual

*Based on a systematic literature review of reports from 1921-2006.

References

  1. Libmeldy, Summary of Product Characteristics.
  2. Rosenberg JB, et al. J Neurosci Res 2016; 94(11): 1169-1179.
  3. Ferreira CR, Gahl WA. Transl Sci Rare Dis 2017; 2(1-2): 1-71.
  4. Gieselmann V, Krägeloh-Mann I. Neuropediatrics. 2010; 41(1): 1-6.
  5. Biffi A et al. Clin Genet. 2008; 74: 349-357.
  6. Wang et al.Wang RY et al. Genet Med. 2011; 13(5): 457-484.
  7. Mahmood A, et al. J Child Neurol. 2010; 25(5): 572-580.
  8. Cesani M et al. Hum Mutat. 2016; 37(1): 16-27.