Orphan Europe files NDA with FDA for Carbaglu (carglumic acid)

Recordati announced the submission to the U.S. FDA by Orphan Europe (a Recordati group company) of an Original New Drug Application (NDA) for Carbaglu (carglumic acid).

The NDA provides for the safe and effective use of Carbaglu in the treatment of hyperammonaemia due to an autosomal genetic disorder resulting in the deficiency of the liver enzyme N-acetylglutamate synthase (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 lifethreatening 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.

In Europe, where sales in 2008 are expected to be of around € 6 million, Carbaglu was granted Orphan Drug status in 2000. The product was granted Orphan Drug status in the USA in 1998 and the FDA granted Carbaglu a Fast Track designation in May 2007 as it is intended to treat a serious or life-threatening condition and address an unmet medical need.