Pegvisomant is the first clinically available growth hormone receptor antagonist. Binding of growth hormone to the growth hormone receptor dimer is necessary for signal transduction to occur. Pegvisomant acts by inhibiting functional growth hormone receptor dimerization. Pegvisomant is currently available for treatment of acromegaly. This rare disease is virtually always caused by a growth hormone-producing pituitary adenoma. In terms of insulin-like growth factor-I normalization, it is the most effective single drug available. However, its current place in the treatment algorithm is after other more established treatment modalities such as surgery, somatostatin analogues and dopamine agonists, have failed. Other potential indications for pegvisomant are treatment and/or prevention of long-term diabetic complications and cancer. These possible indications should be thoroughly investigated as they are very prevalent disorders and even a small positive effect might prove very efficacious on a population level.

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doi.org/10.1586/14750708.2.4.525, hdl.handle.net/1765/61400
Therapy
Department of Internal Medicine

Muller, A., & van der Lely, A.-J. (2005). Pegvisomant: Current and possible future indications. Therapy (Vol. 2, pp. 525–532). doi:10.1586/14750708.2.4.525