Carbaglu

carglumic acid

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This is a summary of the European public assessment report (EPAR) for Carbaglu. 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 Carbaglu.

What is Carbaglu?

Carbaglu is a medicine that contains the active substance carglumic acid. It is available as dispersible tablets. ‘Dispersible’ means that the tablets can be dispersed (mixed) in water.

What is Carbaglu used for?

Carbaglu is used for the treatment of hyperammonaemia (high blood levels of ammonia) in patients with the following metabolic diseases:

  • N-acetylglutamate synthase (NAGS) deficiency. Patients with this lifelong disease lack a liver enzyme called NAGS, which normally helps to break down ammonia. If the enzyme is not present, ammonia cannot be broken down and it builds up in the blood;
  • some organic acidaemias (isovaleric acidaemia, methylmalonic acidaemia and propionic acidaemia) where patients lack certain enzymes involved in protein metabolism.

Because the number of patients with these diseases is low, they are considered ‘rare’ and Carbaglu was designated an ‘orphan medicine’ (a medicine used in rare diseases) on various dates (see below).

The medicine can only be obtained with a prescription.

How is Carbaglu used?

Carbaglu treatment should be started by a doctor who has experience in treating patients with metabolic diseases.

In patients with NAGS deficiency, treatment may be started as early as the first day of life and the medicine is used for the patient’s whole life. In patients with organic acidaemias, treatment is started when the patient has a hyperammonaemia crisis and continued until the crisis is finished.

The initial daily dose of Carbaglu should be 100 mg per kilogram body weight, but up to 250 mg/kg can be used if necessary. The dose should then be adjusted to maintain normal blood ammonia levels. The tablets should be dispersed (mixed) in a small amount of water before being given to the patient. They can easily be broken into two equal halves.

How does Carbaglu work?

When ammonia builds up in the blood, it is toxic to the body, especially the brain. Carbaglu is very similar in structure to N-acetylglutamate, which activates an enzyme that breaks down ammonia. Carbaglu therefore helps break down ammonia, reducing ammonia blood levels and its toxic effects.

How has Carbaglu been studied?

Carbaglu has been studied in 20 patients, 12 of whom had NAGS deficiency and were treated for an average of about three years. The other eight patients were treated for hyperammonaemia of another cause. The company also presented information from the published literature on a further four patients treated with the active substance in Carbaglu.

Carbaglu has also been studied in 57 patients (about two-thirds were newborn babies) with isovaleric acidaemia, methylmalonic acidaemia or propionic acidaemia who were treated with Carbaglu during hyperammonaemia crises.

In all of the studies, the main measure of effectiveness was the change in ammonia levels in the blood.

What benefit has Carbaglu shown during the studies?

In patients with NAGS deficiency, ammonia levels were brought back to normal after Carbaglu treatment. Patients on Carbaglu could be kept stable without a need for restrictions to the diet or the use of other medicines.

In patients with isovaleric acidaemia, methylmalonic acidaemia or propionic acidaemia, Carbaglu also induced a decrease in ammonia levels in the blood, following an average of 5.5 days of treatment.

What is the risk associated with Carbaglu?

The most common side effect with Carbaglu (seen in between 1 and 10 patients in 100) is increased sweating. For the full list of side effects reported with Carbaglu, see the package leaflet.

Carbaglu should not be used in people who may be hypersensitive (allergic) to carglumic acid or any of the other ingredients. Carbaglu must not be used in women who are breastfeeding.

Why has Carbaglu been approved?

The CHMP concluded that Carbaglu was effective in reducing blood ammonia to normal levels and decided that Carbaglu’s benefits are greater than its risks. The Committee recommended that Carbaglu be given marketing authorisation.

Other information about Carbaglu

The European Commission granted a marketing authorisation valid throughout the European Union for Carbaglu to Orphan Europe on 24 January 2003. The marketing authorisation is valid for an unlimited period.

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

Name Language First published Last updated
Carbaglu : EPAR - Summary for the public BG = bălgarski 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public ES = español 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public CS = čeština 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public DA = dansk 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public DE = Deutsch 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public ET = eesti keel 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public EL = elliniká 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public EN = English 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public FR = français 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public IT = italiano 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public LV = latviešu valoda 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public LT = lietuvių kalba 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public HU = magyar 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public MT = Malti 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public NL = Nederlands 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public PL = polski 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public PT = português 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public RO = română 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public SK = slovenčina 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public SL = slovenščina 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public FI = suomi 08/04/2009 19/07/2011
Carbaglu : EPAR - Summary for the public SV = svenska 08/04/2009 19/07/2011

This EPAR was last updated on 16/07/2014 .

Authorisation details

Product details

Product details for Carbaglu
NameCarbaglu
Agency product numberEMEA/H/C/000461
Active substance

carglumic acid

International non-proprietary name (INN) or common name

carglumic acid

Therapeutic area Amino Acid Metabolism, Inborn ErrorsPropionic Acidemia
Anatomical therapeutic chemical (ATC) code A16AA05
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.

Publication details

Publication details for Carbaglu
Marketing-authorisation holder

Orphan Europe S.A.R.L.

Revision10
Date of issue of marketing authorisation valid throughout the European Union24/01/2003

Contact address:

Orphan Europe SARL
Immeuble "Le Wilson"
70, avenue du Général de Gaulle
FR-92800 Puteaux
France

Product information

Product information

19/12/2013  Carbaglu -EMEA/H/C/000461 -IG/0392

Name Language First published Last updated
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014
Carbaglu : EPAR - Product Information SV = svenska 21/09/2009 16/07/2014

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.



Pharmacotherapeutic group

Other alimentary-tract and metabolism products

Therapeutic indication

Carbaglu is indicated in treatment of:

  • hyperammonaemia due to N-acetylglutamate-synthase primary deficiency;
  • hyperammonaemia due to isovaleric acidaemia;
  • hyperammonaemia due to methymalonic acidaemia;
  • hyperammonaemia due to propionic acidaemia.

Assessment History

Changes since initial authorisation of medicine

Initial marketing-authorisation documents

Name Language First published Last updated
Carbaglu : EPAR - Procedural steps taken before authorisation SV = svenska 09/11/2006  
Carbaglu : EPAR - Scientific Discussion SV = svenska 09/11/2006  

Authorised

This medicine is approved for use in the European Union

More information on Carbaglu

This product originally had an orphan designation for the treatment of n-acetylglutamate synthetase (NAGS) deficiency, granted on 18 October 2000. This designation was withdrawn from the Community register of orphan medicinal products in January 2013 at the end of the 10-year period of market exclusivity.