The European Commission has approved Novartis’ drug Signifor as a treatment for the endocrine disorder Cushing’s disease where surgery is impossible or has failed.
Signifor (pasireotide) is the first targeted treatment for Cushing’s disease, in which a benign pituitary tumour triggers excess production of the hormone cortisol with debilitating effects.
The approval followed a phase III clinical trial in which patients given Signifor by regular injection experienced a sustained fall in cortisol levels, with normalisation achieved in a distinct patient sub-group.
Cushing’s disease is a rare but serious condition that affects one to two people per million each year. Symptoms include obesity, weakness and hypertension.
First-line treatment is surgical removal of the pituitary gland, but that is not always safe or successful.
Signifor already has orphan drug designation for treatment of Cushing’s disease.
In the PASPORT-CUSHINGS clinical trial, the largest phase III study of a treatment for Cushing’s disease, 162 patients received Signifor 900ug or 600ug twice daily for six months.
Mean urinary-free cortisol (UFC) levels were normalised in 26.3% and 14.6% of the patients respectively. The former patient group was judged to have achieved the primary trial endpoint of normalisation.
Reductions in UFC were rapid and sustained for both patient groups, with most patients experiencing a decrease within two months. Reductions in clinical symptoms were observed after six months in both patient groups, especially in those patients with normalised UFC levels.
“Patients with Cushing’s disease often struggle with a variety of debilitating health issues and previously were faced with a treatment approach limited to surgery,” said Ellen van Veldhuizen, board member of the Dutch Adrenal Society. “The approval of pasireotide as a new treatment option that may help patients with Cushing’s disease is welcome news.”
Herve Hoppenot, President of Novartis Oncology, commented: “As the first therapeutic option to specifically target Cushing’s disease, Signifor has the potential to redefine treatment of this debilitating disease.”