Metreleptin (Myalepta®) for treatment of lipodystrophy

Zorginstituut Nederland has completed its assessment whether metreleptin (Myalepta®) for treatment of lipodystrophy can be accepted into the Medicine Reimbursement System (GVS).

Registered indication

Metreleptin (Myalepta®) is a orphan drug used for:

  • the treatment of patients two years of age and older with confirmed, congenital, generalized lipodystrophy (Berardinelli-Seip syndrome) or acquired generalized lipodystrophy
  • patients 12 years and older with familial partial lipodystrophy or acquired partial lipodystrophy (Barraquer-Simons syndrome) without adequate metabolic control under standard treatment

Lipodystrophy syndromes are clinically heterogeneous inherited or acquired ultra-rare disorders characterised by selective but variable loss of adipose tissue. Deficient adipose mass may result in ectopic lipid storage in the liver, muscle and other organs. Lipodystrophies can be classified according to the distribution of fat loss (generalized or partial) and as congenital or acquired. This yields 4 major categories: congenital generalized lipodystrophy (CGL; or Berardinelli-Seip syndrome), acquired generalized lipodystrophy (AGL; or Lawrence syndrome), familial partial lipodystrophy (FPL; or Kobberling syndrome or Dunnigan syndrome), and acquired partial lipodystrophy (APL; or Barraquer-Simons syndrome). In GL patients, metabolic complications are common and can be severe. Also in FPL patients metabolic complications are common in adulthood. In APL patients metabolic complications are uncommon. Patients with lipodystrophy, especially generalized forms, are typically hyperphagic which makes it difficult to achieve dietary restriction.

BeNeLuxA project

Zorginstituut Nederland (ZIN) and the Belgian Commission Reimbursement of Medicines (CRM) worked together in this evaluation. The evaluation is part of a common evaluation in the context of the BeNeLuxA project. This report, as well as the Budget Impact Analysis will be used both by ZIN and by the CRM. The relative effectiveness report has been prepared by the Belgian Rijksinstituut voor Ziekte- en invaliditeitsverzekering (RIZIV), the Budget Impact Analysis by ZIN. Both assessment procedures are running in parallel according to the national legislation.


Taking into account the uncertainties in the favorable effects and the concern regarding the development of neutralizing antibodies, ZIN and the Belgian CRM conclude it is not possible to conclude on an added value for metreleptin (Myalepta®) in patients with generalized lipodystrophy and partial lipodystrophy. Due to insufficient data it is concluded that metreleptin has no added benefit (‘a lower therapeutic value’) in comparison with standard treatment.
Based on the results of the assessment, Zorginstituut Nederland advises against including metreleptin (Myalepta®) in the GVS.