EU approves Signifor from Novartis
26.04.2012 - Novartis has gained EU marketing approval for the first targeted therapy to treat Cushing’s disease, a rare endocrine disorder.
Basel – Swiss pharma major Novartis has got the green light from the European Commission to market Signifor (pasiretoid) as treatment for Cushing disease, a debilitating endocrine disorder caused by the over-expression of cortisol. Data from a Phase III trial on 162 patients with the disease showed that subcutaneous injection of the somatostatin analogue twice daily normalised the level of cortisol in the urine („urinary-free cortisol, UFC“) by 47,9% after six months of treatment (N Engl J Med. 2012 Mar 8;366(10):914-24). Clinical symptoms such as high blood pressure, high BMI and cholesterol levels and overweight in the two dosage groups (2 x 600µg and 2x 900 µg) significantly improved after administration of the orphan drug. About 250,000 people in the EU suffer from the disease. In two thirds of them, the disorder is caused by so-called pituitary tumours that release the corticoid hormone ACTH which then triggers cholesterol over-expression. Signifor is approved for adult patients with inoperable pituitary tumours. It reduces ACTH levels by targeting the somatostatin receptor 5 with 40-fold higher affinity as any other somatostatin analogue. In January 2012 the European Medicines Agency in London had recommended approval of Signifor as first-in-class treatment for Cushing’s disease.