The somatostatin analogue would become the first approved medical therapy for the condition after it met the primary endpoint of normalising urinary-free cortisol (UFC) levels in a Phase III trial.
Hervé Hoppenot, President, Novartis Oncology, says the company is now “one step closer to being able to offer patients in Europe” a medical treatment.
Cushing’s disease is a debilitating and fatal disease which affects approximately one to two patients per million, per year. There are believed to be around a total of 20,000 people in Europe with the disease.
First-line therapy is the surgical removal of the tumour of the pituitary that produces too much adrenocorticotrophin (ACTH) and stimulates the adrenal glands to both grow and release excessive amounts of cortisol in to the blood. Symptoms include weight gain, bruising and high blood pressure.
Signifor has been designated as an orphan medicine since October 2009. In the Phase III PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio – CUSHING’S disease) trial, the largest randomised study to evaluate a medical therapy in patients with the disease, it was shown to reduce levels of cortisol in urine by more than half in 41% of patients treated with a 900μg dose, and in more than a third of patients treated with a 600μg dose.
The European Commission will now decide whether to adopt the positive opinion of the CHMP. A decision is expected within the next three months and will apply to all 27 EU member states, plus Iceland and Norway.