Development and co-development projects
Pheburane has been developed by Lucane Pharma for the treatment of Urea Cycle Disorders. A urea cycle disorder (UCD) is an inherited, inborn error of metabolism present in an estimated 1 in 10,000 births. The onset may occur at any age depending on the severity of the disorder. If clinical symptoms are present just after birth, however, the disorder is usually very severe and may be fatal.
UCDs are caused by a deficiency of one of the enzymes in the urea cycle which is responsible for the removal of ammonia, a potent neurotoxin, from the body. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is removed from the blood and converted to urea which is excreted in the urine. In UCDs, the nitrogen removal is blocked and it accumulates in the form of ammonia. Left untreated, UCDs can cause dangerously increased levels of ammonia in the bloodstream (hyperammonemia) resulting in brain damage, coma and, if untreated, death.
The current treatment of urea cycle disorders consists of dietary management to limit ammonia production (with an emphasis on protein restriction and amino acid supplementation) in conjunction with medications and/or supplements which provide alternative pathways for the removal of ammonia from the bloodstream. Patients suffering from this very serious inborn error of metabolism are at risk of neurological damage due to a metabolic imbalance or decompensation if they fail to take their medication in large doses every day.
Phenylbutyrate is a standard treatment to remove ammonia. However, this active substance has a very strong bitter and salty taste.
Pheburane is a new formulation of phenylbutyrate where the taste is completely masked. Lucane has received the Marketing Authorisation from the EMA in July 2013
Ucedane has been developed by Lucane Pharma for the treatment of hyperammonaemia due to N-acetylglutamate synthase primary deficiency (NAGS), which is a urea cycle disorder.
The active substance of Ucedane is carglumic acid, an amino acid derivative. It is a structural analogue of N-acetylglutamate and can replace it in patients who cannot synthesise the compound properly due to a deficiency in the enzyme responsible for its synthesis (N-acetylglutamate synthase).
Ucedane is available as 200-mg dispersible tablets and is a generic of Carbaglu. Studies have demonstrated the satisfactory quality of Ucedane, and its bioequivalence to the reference product Carbaglu.