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Somavaratan is a proprietary long-acting recombinant human growth hormone (rhGH) fusion protein developed for the treatment of growth hormone deficiency (GHD) in both children and adults. The drug consists of an active rhGH portion fused to hydrophilic amino acid sequences (XTEN), which prolongs its half-life and allows for less frequent dosing compared to daily rhGH injections. Its mechanism of action is as a somatotropin receptor agonist; it binds to and activates the somatotropin (growth hormone) receptor, stimulating production of insulin-like growth factor 1 (IGF-1), which mediates many effects of GH including increased height velocity in children with GHD. Clinical studies demonstrated that somavaratan could be dosed weekly or even monthly due to its extended duration of action. Despite promising pharmacokinetics and efficacy data in early trials, development was discontinued after phase II/III trials failed to meet efficacy endpoints[1][3][6][7][8].
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