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Mecasermin rinfabate is a recombinant human protein complex consisting of insulin-like growth factor-1 (rhIGF-1) and its primary binding protein, insulin-like growth factor binding protein-3 (rhIGFBP-3). Developed by Insmed and later investigated by companies such as Shire (Takeda) for neonatal indications, the drug is designed to mimic the endogenous IGF-1/IGFBP-3 complex. This binary complex extends the circulating half-life of IGF-1 and modulates its activity, significantly reducing the risk of hypoglycemia compared to IGF-1 administered alone. By binding to the IGF-1 receptor (IGF1R), it promotes systemic growth, muscle differentiation, and protein synthesis. It was originally approved by the FDA in 2005 for children with severe primary IGF-1 deficiency but was later withdrawn from the US market for growth-related indications due to patent litigation. It has since been evaluated in clinical trials for various conditions, including Noonan syndrome, myotonic dystrophy type 1 (DM1), severe burns, and complications of prematurity such as retinopathy of prematurity (ROP) and bronchopulmonary dysplasia (BPD).
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