Swiss drug giant Novartis AG (NVS) said Wednesday a panel of the US Food and Drug Administration has recommended approval of Signifor (pasireotide) as the first medication to treat U.S. patients with Cushing's disease who require therapeutic intervention.
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 about one to two patients per million per year.
The FDA's Endocrinologic and Metabolic Drugs Advisory Committee voted unanimously in support of Signifor, based on trial data which showed that mean urinary-free cortisol (UFC), the key measure of biochemical control of the disease, was rapidly decreased and sustained in most patients.
The FDA usually considers the recommendations of its panel even as it is not bound to do so.
The most reported adverse events for pasireotide were diarrhea, nausea, hyperglycemia, cholelithiasis, abdominal pain, diabetes mellitus, injection site reactions, among others.
Pasireotide is a multireceptor targeting somatostatin analog that binds with high affinity to four of the five somatostatin receptor subtypes. In April 2012, the European Commission approved pasireotide under the brand name Signifor for the treatment of adult patients with Cushing's disease for whom surgery is not an option or for whom surgery has failed. Other worldwide regulatory filings for pasireotide for this use are also underway.
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