Myalepta is a medicine used in addition to diet to treat lipodystrophy, where patients have loss of fatty tissue under the skin and build-up of fat elsewhere in the body such as in the liver and muscles. The medicine is used in:
- adults and children above the age of 2 years with generalised (throughout the body) lipodystrophy (Berardinelli-Seip syndrome and Lawrence syndrome);
- adults and children above the age of 12 years with partial (localised) lipodystrophy (including Barraquer-Simons syndrome), when standard treatments have failed.
Myalepta contains the active substance metreleptin.
Because the number of patients with the various forms of lipodystrophy is low, the diseases are considered ‘rare’, and Myalepta was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 17 July 2012. Further information on the orphan designations can be found on the European Medicines Agency’s website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation (Bernardinelli-Seip syndrome: 17/07/12; Lawrence syndrome: 17/07/12; familial partial lipodystrophy: 17/07/12; Barraquer-Simons syndrome: 17/07/12).
Myalepta : EPAR - Medicine overview (PDF/84.64 KB)
First published: 06/08/2018
Last updated: 06/08/2018
Myalepta : EPAR - Risk-management-plan summary (PDF/82.11 KB)
First published: 06/08/2018
Last updated: 20/11/2020
|Agency product number||
|International non-proprietary name (INN) or common name||
|Therapeutic area (MeSH)||
Lipodystrophy, Familial Partial
|Anatomical therapeutic chemical (ATC) code||
This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.
Amryt Pharmaceuticals DAC
|Date of issue of marketing authorisation valid throughout the European Union||
26/11/2020 Myalepta - EMEA/H/C/004218 - IAIN/0016/G
- Annex I - Summary of product characteristics
- Annex IIA - Manufacturing-authorisation holder responsible for batch release
- Annex IIB - Conditions of the marketing authorisation
- Annex IIIA - Labelling
- Annex IIIB - Package leaflet
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Other alimentary tract and metabolism products
Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients:
- with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above
- with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control.