Mecasermin.

Mecasermin (recombinant human insulin-like growth factor-I [IGF-I]) is approved in the US for the long-term treatment of growth failure in children with severe primary IGF-I deficiency or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH, and in the EU for the long-term treatment of growth failure in children and adolescents with severe primary IGF-I deficiency. Subcutaneous mecasermin 0.12 mg/kg twice daily stimulated linear growth in children with growth failure and severe IGF-I deficiency associated with GH insensitivity, according to the results of a noncomparative, multicenter trial (n = 76) [mean duration of therapy 4.4 years; range 0.04-12.5 years]. During the first year of treatment, height velocity significantly increased from a mean 2.8 cm/year at baseline to a mean 8.0 cm/year; mean growth velocities remained above baseline for up to 8 years. Mecasermin also promoted statural growth in a small noncomparative trial in children with growth failure and GH insensitivity syndrome (n = 8). After 6.5-7.5 years of mecasermin therapy, the mean increase in the height standard deviation score was +1.4. Mecasermin was also shown to have beneficial effects in various other conditions including diabetes mellitus and anorexia nervosa. Subcutaneous mecasermin was generally well tolerated in children with severe IGF-I deficiency associated with GH insensitivity.