FDA Approved Growth Hormone Axis

Pasireotide

also known as: Signifor, Signifor LAR, SOM230

The somatostatin analog for Cushing's disease — the only FDA-approved medical therapy targeting pituitary ACTH secretion, with uniquely broad somatostatin receptor affinity.

A cyclohexapeptide somatostatin analog with 40-fold higher affinity for sst5 than octreotide, FDA-approved for Cushing's disease (the only approved medical therapy targeting the pituitary) and acromegaly.

Mechanism of action

Pan-somatostatin receptor agonist with highest affinity for sst5 (40x octreotide), and strong binding at sst1, sst2, sst3. In Cushing's disease, sst5 activation on corticotroph adenoma cells suppresses ACTH secretion. In acromegaly, sst2/sst5 co-activation suppresses GH more broadly than octreotide. Hyperglycemia is a major side effect (sst5-mediated suppression of insulin).

Primary uses

Cushing's disease (pituitary ACTH suppression)
Acromegaly (octreotide-refractory)
Neuroendocrine tumor research

Typical dosing

0.3–0.9 mg twice daily (SC) or 40–60 mg monthly (LAR IM) (subcutaneous or intramuscular)

Hyperglycemia occurs in ~70% of patients; glucose monitoring required. May require diabetes management.

Regulatory status

FDA-approved in 2012 (Signifor, Recordati/Novartis) for Cushing's disease in patients not candidates for surgery. LAR formulation approved in 2014 for acromegaly inadequately controlled by surgery or other somatostatin analogs.

References

[clinical-trial] Colao A, et al. "A 12-month phase 3 study of pasireotide in Cushing's disease." N Engl J Med, 2012;366:914-924.
[fda-pi] Signifor (pasireotide) Prescribing Information. Recordati.

Related peptides

Octreotide

The first-generation somatostatin analog (FDA-approved as Sandostatin, 1988) — the pharmacological counterweight to growth-hormone excess. Used for acromegaly, carcinoid syndrome, and VIPoma; now available in injectable, LAR-depot, and oral (Mycapssa, 2020) formulations.

Lanreotide

The second-generation somatostatin analog (FDA-approved as Somatuline Depot, 2007 for acromegaly; 2014 for GEP-NETs; 2017 for carcinoid) — monthly deep-SC prefilled syringe. Functionally equivalent to octreotide LAR for most indications.

Disclaimer

This entry is for educational purposes only and does not constitute medical advice. Dosing information reflects published regulatory or research data and is not a recommendation. Many compounds described here are not approved for human use in the United States. Consult a licensed medical professional before considering any peptide therapy.