Myalepta

metreleptin

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An overview of Myalepta and why it is authorised in the EU

What is Myalepta and what is it used for?

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).

How is Myalepta used?

Myalepta can only be obtained with a prescription and treatment should be started and monitored by a doctor experienced in the diagnosis and management of patients with metabolic disorders.

Myalepta is given as a daily injection under the skin of the abdomen (belly), thigh or upper arm, at the same time every day. The recommended daily dose depends on the patient’s bodyweight and is adjusted based on the patient’s response to treatment. Patients or carers can inject the medicine themselves once they have been trained.

For more information about using Myalepta, see the package leaflet or contact your doctor or pharmacist.

How does Myalepta work?

Patients with lipodystrophy have low levels of a human hormone called leptin, which plays a key role in regulating the break-down of fats and sugars in the body. This leads to loss of fat from under the skin and its build-up in places such as the liver and muscles, as well as high levels of fat in the blood. It also results in insulin resistance (when the body is unable to recognise insulin, a hormone that helps regulate blood sugar levels).

The active substance in Myalepta, metreleptin, is similar to leptin. It replaces leptin and increases fat breakdown in the blood, muscles and liver, thereby correcting some abnormalities in patients with lipodystrophy, including insulin resistance. However, the medicine does not restore fat tissue under the skin.

What benefits of Myalepta have been shown in studies?

Myalepta has been shown to be effective at lowering blood fat levels in 2 main studies involving a total of 107 adults and children with generalised or partial lipodystrophy. In the studies Myalepta was not compared with any other treatment. After 12 months of treatment, blood levels of fats (triglycerides) decreased from around 15 mmol/l to around 5 mmol/l in patients with generalised disease, and from around 16 mmol/l to around 6 mmol/l in patients with partial disease.

Insulin resistance was also improved: blood levels of a substance called glycosylated haemoglobin (HbA1c), which gives an indication of how well the blood glucose is controlled, fell from 8.6% to 6.4% in patients with generalised lipodystrophy, and from 8.8% to 8.0% in patients with partial lipodystrophy.

What are the risks associated with Myalepta?

The most common side effects with Myalepta (which may affect more than 1 in 10 people) are hypoglycaemia (low blood glucose) and weight loss. For the full list of side effects and restrictions with Myalepta, see the package leaflet.

Why is Myalepta authorised in the EU?

Myalepta has been shown to correct some abnormalities caused by leptin deficiency in patients with lipodystrophy, a rare condition for which few treatments are available. Side effects seen with Myalepta are of the kind expected from this type of treatment. The European Medicines Agency therefore decided that Myalepta’s benefits are greater than its risks and it can be authorised for use in the EU.

Myalepta has been authorised under ‘exceptional circumstances’. This is because it has not been possible to obtain complete information about Myalepta due to the rarity of the condition. Every year, the Agency will review any new information that becomes available and this overview will be updated as necessary.

What information is still awaited for Myalepta?

Since Myalepta has been authorised under exceptional circumstances, the company that markets Myalepta will set-up a registry of patients treated the medicine, and conduct studies to further investigate the benefits and risks of treatment including the possibility for Myalepta to trigger the production of antibodies.

What measures are being taken to ensure the safe and effective use of Myalepta?

The company that markets Myalepta will provide educational materials to patients and doctors with detailed information on how to use the medicine and what to do in case of side effects, including allergic reactions, hypoglycaemia and serious infections.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Myalepta have also been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Myalepta are continuously monitored. Side effects reported with Myalepta are carefully evaluated and any necessary action taken to protect patients.

Other information about Myalepta

Myalepta received a marketing authorisation valid throughout the EU on 30 July 2018.

Name Language First published Last updated
Myalepta : EPAR - Medicine overview BG = bălgarski 2018-08-06  
Myalepta : EPAR - Medicine overview ES = español 2018-08-06  
Myalepta : EPAR - Medicine overview CS = čeština 2018-08-06  
Myalepta : EPAR - Medicine overview DA = dansk 2018-08-06  
Myalepta : EPAR - Medicine overview DE = Deutsch 2018-08-06  
Myalepta : EPAR - Medicine overview ET = eesti keel 2018-08-06  
Myalepta : EPAR - Medicine overview EL = elliniká 2018-08-06  
Myalepta : EPAR - Medicine overview EN = English 2018-08-06  
Myalepta : EPAR - Medicine overview FR = français 2018-08-06  
Myalepta : EPAR - Medicine overview IT = italiano 2018-08-06  
Myalepta : EPAR - Medicine overview LV = latviešu valoda 2018-08-06  
Myalepta : EPAR - Medicine overview LT = lietuvių kalba 2018-08-06  
Myalepta : EPAR - Medicine overview HU = magyar 2018-08-06  
Myalepta : EPAR - Medicine overview MT = Malti 2018-08-06  
Myalepta : EPAR - Medicine overview NL = Nederlands 2018-08-06  
Myalepta : EPAR - Medicine overview PL = polski 2018-08-06  
Myalepta : EPAR - Medicine overview PT = português 2018-08-06  
Myalepta : EPAR - Medicine overview RO = română 2018-08-06  
Myalepta : EPAR - Medicine overview SK = slovenčina 2018-08-06  
Myalepta : EPAR - Medicine overview SL = slovenščina 2018-08-06  
Myalepta : EPAR - Medicine overview FI = suomi 2018-08-06  
Myalepta : EPAR - Medicine overview SV = svenska 2018-08-06  
Myalepta : EPAR - Medicine overview HR = Hrvatski 2018-08-06  
Name Language First published Last updated
Myalepta : EPAR - Risk-management-plan summary (English only) 2018-08-06  

This EPAR was last updated on 06/08/2018 .

Authorisation details

Product details

Product details for Myalepta
NameMyalepta
Agency product numberEMEA/H/C/004218
Active substance

metreleptin

International non-proprietary name (INN) or common name

metreleptin

Therapeutic area Lipodystrophy, Familial Partial
Anatomical therapeutic chemical (ATC) code A16AA
Additional monitoring

This medicine is under additional monitoring. This means that it is being monitored even more intensively than other medicines. For more information, see medicines under additional monitoring.

Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Exceptional Circumstances

There were "exceptional circumstances" concerning the approval of this medicine. This happens when the applicant can show that they are unable to provide comprehensive data on the efficacy and safety of the medicine for which authorisation is being sought, due to the rarity of the condition it is intended for, limited scientific knowledge in the area concerned, or ethical considerations involved in the collection of such data.

Publication details

Publication details for Myalepta
Marketing-authorisation holder

Aegerion Pharmaceuticals B.V.


 

Revision0
Date of issue of marketing authorisation valid throughout the European Union30/07/2018

Contact address:

Aegerion Pharmaceuticals B.V.
Atrium Building, 8th Floor
Strawinskylaan 3127
1077 ZX Amsterdam
The Netherlands

Product information

Product information

30/07/2018  Myalepta -EMEA/H/C/004218

Name Language First published Last updated
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  
Myalepta : EPAR - Product Information EN = English 2018-08-06  

Contents

  • 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

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  
Myalepta : EPAR - All Authorised presentations EN = English 2018-08-06  

Pharmacotherapeutic group

Other alimentary tract and metabolism products

Therapeutic indication

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.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated

Initial marketing-authorisation documents

Name Language First published Last updated
Myalepta : Orphan maintenance assessment report EN = English 2018-08-06  
Myalepta : EPAR - Public assessment report EN = English 2018-08-06  
CHMP summary of positive opinion for Myalepta EN = English 2018-06-01  

Authorised

This medicine is approved for use in the European Union

Preventing medication errors