Basic Information
EMA regulatory identification and product classification information
EMA Identifiers
Overview Summary
Comprehensive product overview and regulatory summary
Ucedane is a medicine 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.
Ucedane contains the active substance carglumic acid and is a ‘generic medicine’. This means that Ucedane contains the same active substance and works in the same way as a ‘reference medicine’ already authorised in the European Union (EU) called Carbaglu.
Active Substances (1)
carglumic acid
Documents (8)
Ucedane : EPAR - Public assessment report
June 29, 2017
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Ucedane : EPAR - Product Information
June 29, 2017
DRUG_PRODUCT_INFORMATION
Ucedane : EPAR - All Authorised presentations
June 29, 2017
AUTHORISED_PRESENTATIONS
Ucedane : EPAR - Procedural steps taken and scientific information after authorisation
September 27, 2017
CHANGES_SINCE_INITIAL_AUTHORISATION
Ucedane : EPAR - Summary for the public
June 29, 2017
OVERVIEW_DOCUMENT
CHMP summary of positive opinion for Ucedane
April 20, 2017
INITIAL_MARKETING_AUTHORISATION_DOCUMENTS
Ucedane : EPAR - Public assessment report
June 29, 2017
CHANGES_SINCE_INITIAL_AUTHORISATION
CHMP summary of positive opinion for Ucedane
April 20, 2017
CHANGES_SINCE_INITIAL_AUTHORISATION
Overview Q&A (7)
Question
How is Ucedane used?
Answer
Ucedane is available as dispersible tablets (200 mg) that are to be dispersed (mixed) in a small amount of water. The medicine can only be obtained with a prescription and 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 Ucedane 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.
For more information about using Ucedane, see the package leaflet or contact your doctor or pharmacist.
Question
How does Ucedane work?
Answer
When ammonia builds up in the blood, it is toxic to the body, especially the brain. The active substance in Ucedane, carglumic acid, is very similar in structure to N acetylglutamate, which activates an enzyme that breaks down ammonia. Ucedane therefore helps break down ammonia, reducing ammonia blood levels and its toxic effects.
Question
Why is Ucedane authorised in the EU?
Answer
The European Medicines Agency concluded that, in accordance with EU requirements, Ucedane has been shown to have comparable quality and to be bioequivalent to Carbaglu. Therefore, the Agency’s view was that, as for Carbaglu, the benefits of Ucedane outweigh the identified risk and it can be authorised for use in the EU.
Question
What measures are being taken to ensure the safe and effective use of Ucedane?
Answer
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Ucedane have been included in the summary of product characteristics and the package leaflet.
Question
Other information about Ucedane
Answer
Ucedane received a marketing authorisation valid throughout the EU on 23 June 2017.
Question
What are the benefits and risks of Ucedane?
Answer
Because Ucedane is a generic medicine and is bioequivalent to the reference medicine, its benefits and risks are taken as being the same as the reference medicine’s.
Question
How has Ucedane been studied?
Answer
Studies on the benefits and risks of the active substance in the approved use have already been carried out with the reference medicine, Carbaglu, and do not need to be repeated for Ucedane.
As for every medicine, the company provided studies on the quality of Ucedane. The company also carried out a study that showed that it is ‘bioequivalent’ to the reference medicine. Two medicines are bioequivalent when they produce the same levels of the active substance in the body and are therefore expected to have the same effect.