Recordatoi shares are up this morning after regulators in the USA has given the green light to Carbaglu for a rare genetic blood disorder.

The US Food and Drug Administration (has approved Carbaglu (carglumic acid) for the treatment of acute hyperammonaemia due to the deficiency of the hepatic enzyme N-acetylglutamate synthase (NAGS deficiency). It has also backed the treatment as maintenance therapy for chronic hyperammonaemia due to NAGS deficiency.

The latter involves extremely high plasma levels of ammonia, which leads to permanent and irreversible damage of the central nervous system. It is a lifelong, life-threatening clinical condition but Recordati says that when treatment with Carbaglu is started early, “patients have normal growth and neurological development, and most of them do not need protein dietary restrictions”.

The approval is based on a trial involving 23 people with NAGS which showed that Carbaglu, which is already approved in Europe, reduced blood ammonia within 24 hours and lowered levels to a normal range within three days. However the FDA insisted that the drug should only be administered by a doctor experienced in treating metabolic disorders.

Janet Woodcock, head of the FDA's Center for Drug Evaluation and Research, said “we are very excited that more drugs are being developed to treat very rare but often devastating genetic disorders". She added that “we hope to see continuing progress in this area”.