MedPath
EMA Approval

Carbaglu

A16AA05

carglumic acid

Other alimentary tract and metabolism products

Basic Information

EMA regulatory identification and product classification information

EMA Identifiers

ATC CodeA16AA05
EMA European Classification

Overview Summary

Comprehensive product overview and regulatory summary

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.

Authorisations (1)

EMEA/H/C/000461

Tour Hekla,Tour Hekla,52 avenue du Général de Gaulle,F-92800 Puteaux,France

Authorised

January 24, 2003

Active Substances (1)

carglumic acid

Documents (12)

Carbaglu : EPAR - Scientific Discussion

November 9, 2006

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

Carbaglu : EPAR - Procedural steps taken before authorisation

November 9, 2006

INITIAL_MARKETING_AUTHORISATION_DOCUMENTS

Carbaglu : EPAR - All Authorised presentations

November 9, 2006

AUTHORISED_PRESENTATIONS

Carbaglu : EPAR - Procedural steps taken and scientific information after authorisation

September 20, 2009

CHANGES_SINCE_INITIAL_AUTHORISATION

Carbaglu : EPAR - Risk management plan summary

July 19, 2023

RISK_MANAGEMENT_PLAN_SUMMARY

CHMP post-authorisation summary of positive opinion for Carbaglu

April 14, 2011

CHANGES_SINCE_INITIAL_AUTHORISATION

Carbaglu-H-C-PSUSA-564-201501 : EPAR - Scientific conclusions and grounds recommending the variation to the terms of the marketing authorisation

March 1, 2016

CHANGES_SINCE_INITIAL_AUTHORISATION

Carbaglu-H-C-461-II-0013 : EPAR - Assessment Report - Variation

July 18, 2011

CHANGES_SINCE_INITIAL_AUTHORISATION

Carbaglu : EPAR - Summary for the public

April 7, 2009

OVERVIEW_DOCUMENT

Carbaglu : EPAR - Product Information

September 20, 2009

DRUG_PRODUCT_INFORMATION

Carbaglu : EPAR - Scientific Discussion

November 9, 2006

CHANGES_SINCE_INITIAL_AUTHORISATION

Carbaglu : EPAR - Procedural steps taken before authorisation

November 9, 2006

CHANGES_SINCE_INITIAL_AUTHORISATION

Overview Q&A (9)

Question

How is Carbaglu used?

Answer

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.

Question

How does Carbaglu work?

Answer

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.

Question

Other information about Carbaglu

Answer

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.

Question

How has Carbaglu been studied?

Answer

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.

Question

What benefit has Carbaglu shown during the studies?

Answer

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.

Question

Why has Carbaglu been approved?

Answer

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.

Question

What is Carbaglu?

Answer

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.

Question

What is Carbaglu used for?

Answer

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.

Question

What is the risk associated with Carbaglu?

Answer

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.

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