Pegvisomant: current and potential novel therapeutic applications

Background: Pegvisomant is a genetically engineered molecule, which exhibits specific growth hormone (GH) antagonism by directly interacting with the GH receptor. It is currently licensed for the treatment of acromegaly where surgery and medical therapy with somatostatin analogues have failed. Objective: To delineate the role of pegvisomant in the treatment of acromegaly and its novel application in other areas of clinical medicine where suppression of GH action may be of therapeutic benefit. Methods: A literature review from PubMed- and EMBASE-listed publications and the web-sites of licensing organisations for medicinal products. Conclusion: Pegvisomant is currently used as a second line therapy in the management of acromegaly. It is highly effective in suppressing the metabolic effects of elevated GH levels when used alone or in combination with somatostatin analogues. However, its long term efficacy and safety for this indication has yet to be established. Preliminary data indicate that pegvisomant may have a role in management of type 1 diabetes with beneficial effects on insulin sensitivity and in preventing the progression of microvascular complications. Additional roles as an adjunct to cancer chemotherapy regimens and for the diagnosis of GH deficiency have been proposed, but have yet to be confirmed.