Myalepta
What Myalepta is used for
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.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
- Approved under exceptional circumstances: full data cannot be collected, usually because the disease is very rare.
Key facts
- Brand
- Myalepta
- Active substance
- metreleptin
- Disease area
- Lipodystrophy, Familial Partial
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AA
- Marketing authorisation holder
- Chiesi Farmaceutici S.p.A.
- Authorised in the EU since
- 2018
Official information
In the USA
Same active substance: Myalept
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Myalepta used for?
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.
What is the active substance in Myalepta?
Myalepta contains metreleptin.
Who makes Myalepta?
The EU marketing authorisation holder is Chiesi Farmaceutici S.p.A..
Is Myalepta an orphan medicine?
Yes. Myalepta is designated an orphan medicine in the EU, for a rare disease (Lipodystrophy, Familial Partial).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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