Recordati announced the approval by the Food and Drug Administration (FDA) in the US of the NDA submitted by Orphan Europe for the use of Carbaglu (carglumic acid) in paediatric and adult patients for the treatment of acute hyperammonaemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS deficiency) and as maintenance therapy for chronic hyperammonaemia due to NAGS deficiency.

NAGS deficiency, a very rare disease involving extremely high plasma levels of ammonia, which leads to permanent and irreversible damage of the central nervous system, is a lifelong serious life-threatening clinical condition. The symptoms start shortly after birth, rapidly leading to cerebral oedema, coma and eventually death without appropriate treatment. Rapid diagnosis and prompt effective treatment are essential to prevent patients from permanent neurological damage.

Carbaglu is the only specific treatment of hyperammonaemia due to NAGS deficiency. Other available treatments are unspecific for this indication. When treatment with Carbaglu is started early, patients have normal growth and neurological development, and most of them do not need protein dietary restrictions. Carbaglu does not only save patients’ lives, but also assures a good quality of life for patients on a continuous treatment.

“We are very pleased with the US FDA’s timely decision to approve Carbaglu and thus make this drug available for patients in the US suffering from such a serious condition,” stated Giovanni Recordati, Chairman and CEO. “This also represents a significant step in the development of our business dedicated to the treatment of rare and orphan diseases.”

Recordati, established in 1926, is a European pharmaceutical group.