Glybera

alipogene tiparvovec

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This is a summary of the European public assessment report (EPAR) for Glybera. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Glybera.

What is Glybera?

Glybera is a medicine that contains the active substance alipogene tiparvovec. It is available as a solution for injection.

Glybera is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

What is Glybera used for?

Glybera is used to treat adults with lipoprotein lipase deficiency who have severe or multiple attacks of pancreatitis (inflammation of the pancreas) despite maintaining a low-fat diet.

Lipoprotein lipase deficiency is a rare disease in which patients have a defect in the gene for lipoprotein lipase, an enzyme responsible for breaking down fats. Patients with this disease need to be on a strict low-fat diet and are prone to recurring attacks of pancreatitis, which is a severe and life-threatening complication.

Glybera is only for patients whose disease has been confirmed by appropriate genetic testing and who have detectable levels of the lipoprotein lipase enzyme in their blood.

Because the number of patients with lipoprotein lipase deficiency is low, the disease is considered ‘rare’, and Glybera was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 March 2004.

The medicine can only be obtained with a prescription.

How is Glybera used?

Glybera should only be prescribed and given under the supervision of a doctor with expertise in treating lipoprotein lipase deficiency and in gene therapy.

Glybera is given as a single treatment involving multiple injections into the muscles of the upper and lower legs. The amount of the medicine to inject and the number of injections depend on the patient’s weight. For more information on the dose of Glybera to be given, see the summary of product characteristics (also part of the EPAR).

For three days before Glybera treatment and for twelve weeks after, patients are given immunosuppressive treatment to reduce the reaction of the body’s immune system against the medicine. Due to the multiple injections required, it is advisable to give it with a spinal or regional anaesthetic (medicines which numb certain parts of the body to block pain).

How does Glybera work?

The active substance in Glybera, alipogene tiparvovec, is derived from a virus that has been modified so it can carry the lipoprotein lipase gene into the body’s cells. When injected into the muscles, it corrects the lipoprotein lipase deficiency by enabling the muscle cells to produce the enzyme. The enzyme produced by these cells can then help to break down fats in the blood, reducing the number of pancreatitis attacks and the severity of the disease.

The modified viral material used in Glybera does not cause infections and cannot make copies of itself.

How has Glybera been studied?

Glybera has been studied in 27 patients with lipoprotein lipase deficiency on a low-fat diet. The majority of patients who received Glybera also received immunosuppressive treatment. The main measures of effectiveness were the reduction in blood fat levels after meals and the reduction in the number of pancreatitis attacks.

What benefit has Glybera shown during the studies?

The data showed a reduction in blood fat levels after meals in some patients. There was also a reduction in the number of pancreatitis attacks in some patients, as well as fewer hospital admissions and stays in intensive care units. Although there were data for only a small number of patients, the results indicate that Glybera would be of benefit to those patients with severe or multiple pancreatitis attacks.

What is the risk associated with Glybera?

The most common side effect reported with Glybera was pain in the legs following the injections, which occurred in a third of patients. Other common side effects reported include headache, tiredness, hyperthermia (high body temperature), contusion (bruising) and increased levels of the enzyme creatine kinase in the blood (a measure of damage to muscle tissue). One patient was diagnosed with pulmonary embolism (clots in blood vessels supplying the lungs) seven weeks after treatment. Given the small number of patients treated, these side effects do not provide a complete picture of the frequency and nature of the side effects of Glybera. For the full list of all side effects reported with Glybera, see the package leaflet.

Glybera must not be used in patients with immunodeficiency (weakened immunity), increased risk of bleeding and muscle disease. Anticoagulant medicines, which can increase bleeding, must not be used from the week before Glybera treatment to one day after. Patients must also not use oral contraceptives. For the full list of restrictions, see the package leaflet.

Why has Glybera been approved?

After careful consideration of all the evidence and the circumstances of the disease, including its extreme rarity, the CHMP concluded that the results from studies showed that the benefits of Glybera outweigh its risks in patients with severe or multiple pancreatitis attacks despite following a low-fat diet. This is a subgroup of severely affected patients with a high unmet medical need. The CHMP therefore recommended that the medicine be granted marketing authorisation.

Glybera has been authorised under ‘exceptional circumstances’ as it has not been possible to obtain complete information about the medicine, because of the rarity of the disease. Every year, the European Medicines Agency will review any new information that becomes available and this summary will be updated as necessary.

What information is still awaited for Glybera?

Under the terms of the authorisation, the company is required to provide further data on fat levels in the blood after meals and on the immune response to Glybera in new patients. The company will also provide data from a registry to monitor the outcome of patients treated with Glybera, and will add a step to the manufacturing of the product to improve the safety profile.

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

A risk management plan has been developed to ensure that Glybera is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Glybera, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company has put in place a restricted access programme to ensure that Glybera is used appropriately when the diagnosis is confirmed. The medicine will only be supplied to doctors who have received the appropriate educational materials and will only be used to treat patients participating in the registry. The company will also provide patients and healthcare professionals with educational materials, including information on how to administer Glybera and how to manage risks with the medicine. Patients will also be provided with a patient alert card.

Other information about Glybera

The European Commission granted a marketing authorisation valid throughout the European Union for Glybera on 25 October 2012.

For more information about treatment with Glybera, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Name Language First published Last updated
Glybera : EPAR - Summary for the public BG = bălgarski 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public ES = español 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public CS = čeština 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public DA = dansk 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public DE = Deutsch 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public ET = eesti keel 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public EL = elliniká 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public EN = English 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public FR = français 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public IT = italiano 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public LV = latviešu valoda 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public LT = lietuvių kalba 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public HU = magyar 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public MT = Malti 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public NL = Nederlands 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public PL = polski 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public PT = português 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public RO = română 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public SK = slovenčina 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public SL = slovenščina 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public FI = suomi 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public SV = svenska 29/11/2012 15/06/2016
Glybera : EPAR - Summary for the public HR = Hrvatski 29/11/2012 15/06/2016

This EPAR was last updated on 15/06/2016 .

Authorisation details

Product details

Product details for Glybera
NameGlybera
Agency product numberEMEA/H/C/002145
Active substance

alipogene tiparvovec

International non-proprietary name (INN) or common name

alipogene tiparvovec

Therapeutic area Hyperlipoproteinemia Type I
Anatomical therapeutic chemical (ATC) code C10 AX10
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 Glybera
Marketing-authorisation holder

uniQure biopharma B.V.
 

Revision7
Date of issue of marketing authorisation valid throughout the European Union25/10/2012

Contact address:

uniQure biopharma B.V.
Meibergdreef 61
NL-1105 BA Amsterdam
The Netherlands

Product information

Product information

28/01/2016  Glybera -EMEA/H/C/002145 -II/0047

Name Language First published Last updated
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016
Glybera : EPAR - Product Information HR = Hrvatski 29/11/2012 22/03/2016

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
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  
Glybera : EPAR - All Authorised presentations HR = Hrvatski 29/11/2012  

Name Language First published Last updated
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  
Glybera : EPAR - Conditions imposed on member states for safe and effective use HR = Hrvatski 29/11/2012  

Pharmacotherapeutic group

Lipid modifying agents

Therapeutic indication

Glybera is indicated for adult patients diagnosed with familial lipoprotein lipase deficiency (LPLD) and suffering from severe or multiple pancreatitis attacks despite dietary fat restrictions. The diagnosis of LPLD has to be confirmed by genetic testing. The indication is restricted to patients with detectable levels of LPL protein.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Glybera : EPAR - Procedural steps taken and scientific information after authorisation HR = Hrvatski 03/09/2014 22/03/2016

Initial marketing-authorisation documents

Authorised

This medicine is approved for use in the European Union

More information on Glybera