What is Ucedane - Carglumic Acid and what is it used for?
Ucedane is a medicine used to treat hyperammonemia (high blood ammonia levels) in patients with N-acetylglutamate synthase (NAGS) deficiency. Patients with this chronic disease show a deficiency of NAGS, which is an enzyme present in the liver that normally contributes to degrade ammonia. If this enzyme is not present, ammonia cannot be degraded and accumulates in the blood.
Ucedane contains the active substance carglumic acid and is a "generic medicine". This means that Ucedane contains the same active substance and acts in the same way as a 'reference medicine', already authorized in the European Union (EU) called Carbaglu. For more information on generic medicines, see the questions and answers by clicking here.
How is Ucedane - Carglumic Acid used?
Ucedane is available as dispersible tablets (200 mg) which must be dispersed (mixed) in a little water. The medicine can only be obtained with a prescription and treatment should be started by a doctor experienced in treating patients with metabolic diseases such as NAGS deficiency.
The treatment can be started from birth and the medicine continues during the patient's entire life.
The initial daily dose of Ucedane should be 100 mg per kilogram of body weight; if necessary the dosage can be increased up to 250 mg / kg. Thereafter, the dose should be adjusted to maintain normal blood ammonia levels.
How does Ucedane - Carglumic Acid work?
When ammonia accumulates in the blood, it becomes toxic to the body and to the brain in particular. The active ingredient of Ucedane, carglumic acid, has a structure very similar to N-acetylglutamate, which activates an enzyme that degrades ammonia. Ucedane, therefore, favors the degradation of ammonia, reducing its concentration in the blood and, consequently, its toxic effects.
What are the benefits and risks of Ucedane - Carglumic Acid?
Studies on the benefits and risks of the active ingredient for the approved use have already been carried out with the reference medicine, Carbaglu, and should therefore not be repeated for Ucedane.
As with any medicine, the company has made studies on the quality of Ucedane available. In addition, he performed a study that showed his "bioequivalence" with the reference medicine. Two medicines are bioequivalent when they produce the same levels of active ingredient in the body, so they are expected to have the same effect.
Why has Ucedane - Carglumic Acid been approved?
Because Ucedane is a generic medicine and is bioequivalent to the reference medicine, its benefits and risks are considered to be the same as those of the reference medicine.
What measures are being taken to ensure the safe and effective use of Ucedane - Carglumic Acid?
Because Ucedane is a generic medicine and is bioequivalent to the reference medicine, its benefits and risks are considered to be the same as those of the reference medicine.
More information on Ucedane - Carglumic Acid
The full EPAR for Ucedane is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information on Ucedane therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist. The full EPAR for the reference medicine can also be found on the Agency's website.
