Wednesday, November 7, 2012

FDA Advisory Committe Recommends Signifor (pasireotide) for approval to treat patients with Cushing's disease

The USFDA Endocrinologic and Metabolic Drugs advisory committee voted unanimously in favor of Signifor as the first medication for the treatment of cushing's disease.  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.

Efficacy of Signifor

Results from the PASPORT-CUSHINGS study found that mean urinary-free cortisol (UFC), the key measure of biochemical control of the disease, was rapidly decreased and sustained in a majority of patients, with a subset of patients reaching normalized levels.  The study also showed that, on average, as UFC levels were reduced, clinical manifestations of Cushing's disease improved.

Safety Profile of Signifor

The most frequently reported adverse events (AEs) (>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 safety profile of pasireotide was similar to that of other somatostatin analogs (SSA) with the exception of the greater degree of hyperglycemia 

 About Cushing Syndrome

Cushing's syndrome is an endocrine disorder caused by excessive cortisol, a vital hormone that regulates metabolism, maintains cardiovascular function and helps the body respond to stress. Cushing's disease is a form of Cushing's syndrome, in which excess cortisol production is triggered by an adrenocorticotropic hormone (ACTH)-secreting pituitary adenoma. It is a rare but serious disease that affects approximately one to two patients per million per year. Cushing's disease most commonly affects adults as young as 20 to 50 years and affects women three times more often than men. It may present with weight gain, central obesity, a round, red full face, severe fatigue and weakness, striae (purple stretch marks), high blood pressure, depression and anxiety. The first line and most common treatment approach for Cushing's disease is surgical removal of the tumor.


About pasireotide / Signifor 

Pasireotide is a multireceptor targeting somatostatin analog (SSA) that binds with high affinity to four of the five somatostatin receptor subtypes (sst 1, 2, 3 and 5)[2]. In April 2012, the European Commission approved pasireotide under the brand name Signiforfor the treatment of adult patients with Cushing's disease for whom surgery is not an option or for whom surgery has failed. 




Enter your email address:


Delivered by FeedBurner