Date: 2012-05-02
Type of information: Granting of a Market Authorisation in the US
Product name: Signifor®
Compound: pasireotide
Therapeutic area:
Action mechanism: Pasireotide, an investigational multireceptor targeting somatostatin analog (SSA), binds with high affinity to four of the five somatostatin receptor subtypes (sst 1, 2, 3 and 5).
Company: Novartis (Switzerland)
Disease: Cushing's disease
Latest news: * On January 20, 2012, The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for Signifor® (SOM230, pasireotide) for the treatment of Cushing's disease. The CHMP positive opinion is based on data from the Phase III PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio - CUSHING'S disease) trial, the largest randomized study to evaluate a medical therapy in patients with Cushing's disease . In the study, patients were randomized to receive pasireotide subcutaneous (sc) injection in doses of 900µg and 600µg twice daily. For the 900µg group, the study met the primary endpoint of normalizing urinary-free cortisol (UFC) levels, the key measure of biochemical control of the disease. Urinary-free cortisol levels were normalized in 26.3% and 14.6% of patients randomized to receive pasireotide 900µg and 600µg twice daily, respectively, at six months of treatment. After 12 months of treatment, results confirmed the durability of the effect. On average, as UFC levels were reduced, clinical manifestations of Cushing\'s disease improved including reduction of blood pressure, total cholesterol, weight and body mass index. The most frequently reported adverse events (AE) (>10%) by investigators for pasireotide were diarrhea, nausea, hyperglycemia, cholelithiasis, abdominal pain, diabetes mellitus, injection site reactions, fatigue and increased glycosylated hemoglobin (HbA1c), with most events being Grade 1-2. The tolerability profile of pasireotide was similar to that of other somatostatin analogs with the exception of the greater degree of hyperglycemia.
* On April 25, 2012, Novartis announced that the European Commission has approved Signifor® (pasireotide) for the treatment of adult patients with Cushing's disease for whom surgery is not an option or for whom surgery has failed. Signifor® is the first medicine to be approved in the European Union (EU) targeting Cushing's disease.
* On November 7, 2012, the FDA Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) has voted unanimously in support of the use of Signifor® (pasireotide) for the treatment of patients with Cushing's disease who require medical therapeutic intervention. The recommendation was based on data from clinical trials of pasireotide, including PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio - CUSHING'S disease), the largest randomized Phase III study to evaluate a medical therapy in patients with Cushing's disease.
* On December 14, 2012, the FDA has approved Signifor® (pasireotide) injection for the treatment of adult patients with Cushing's disease for whom pituitary surgery is not an option or has not been curative3. Signifor® is the first medicine to be approved in the US that addresses the underlying mechanism of Cushing's disease.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2012-12-14
UE authorization: 2012-04-25
Favourable opinion UE: 2012-01-20
Favourable opinion USA: 2012-11-07
Orphan status USA: 2009-07-24
Orphan status UE: 2009-10-08
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
