Pasireotide (Signifor): Uses, Evidence, and Side Effects

Key takeaways

• Pasireotide is a somatostatin analog approved under the brand name Signifor.
• It is indicated for adults with Cushing's disease when surgery is not an option or has not worked.
• The drug acts by binding somatostatin receptors, targeting a receptor subtype often overexpressed in Cushing's disease tumor cells.
• Across 87 registered trials, pasireotide has also been studied in neuroendocrine tumors and dumping syndrome.
• The most reported adverse events involve blood sugar changes, alongside gastrointestinal and general symptoms.

What Pasireotide Is

Pasireotide is an injectable cyclohexapeptide classified as a somatostatin analog. It works by binding to somatostatin receptors, a family of five receptor subtypes found throughout the body. Corticotroph tumor cells in patients with Cushing's disease frequently overexpress one of these subtypes, SSTR5, which the drug is designed to engage. By binding these receptors, pasireotide affects hormone signaling patterns linked to the underlying disease.

What Pasireotide Is Approved For

Pasireotide, sold as Signifor, is approved to treat adult patients with Cushing's disease. It is specifically indicated for those for whom pituitary surgery is not an option or has not achieved a cure. The label does not carry a boxed warning.

What The Evidence Shows

Pasireotide has been studied across 87 registered clinical trials covering multiple conditions. One phase 2 trial examined pasireotide combined with everolimus for advanced pancreatic neuroendocrine tumors, though it was terminated. A separate completed phase 2 study evaluated pasireotide's efficacy, safety, and pharmacokinetics in patients with dumping syndrome.

Reported Side Effects

Data from the FDA Adverse Event Reporting System reflect report counts, not confirmed incidence or direct causation. The most frequently reported reactions relate to blood sugar disruption, including increased blood glucose (259 reports), hyperglycaemia (247), and diabetes mellitus (244). Gastrointestinal and general symptoms were also common, with nausea (228), diarrhoea (209), fatigue (203), and headache (175) among the top reported terms. A notable share of reports (172) were coded as off label use.