Definition

An antisense oligonucleotide targeting the growth hormone receptor (GHR; somatotropin receptor) mRNA, with potential somatotropin-inhibiting activity. Upon subcutaneous administration, cimdelirsen hybridizes with GHR mRNA, which blocks translation of the GHR protein. Reduction of GHR levels in turn decreases the circulating level of insulin-like growth factor-1 (IGF-1). IGF-1 is a hormone that plays an important role in childhood growth and has anabolic effects in adults. Excess production of IGF-1 results in various disease states including acromegaly.