Mecasermin (rhIGF-1) long-term safety and effectiveness evaluated in children with Primary IGF-1 Deficiency

Primary insulin-like growth factor-1 deficiency (Primary IGFD) is a rare genetic disorder characterized by severe growth failure and short stature, distinct from growth hormone deficiency. In Primary IGFD, the body either produces insufficient IGF-1 or has impaired IGF-1 receptor signaling, despite adequate growth hormone (GH) levels. Current treatments for growth failure, such as recombinant human growth hormone (rhGH), are ineffective in these patients because their underlying issue is a lack of IGF-1, not GH. Therefore, direct replacement therapy with recombinant human IGF-1 (rhIGF-1), like mecasermin, is crucial to bypass the defective GH-IGF-1 axis and promote normal growth and development. Long-term data on its safety and efficacy are vital for pediatric populations.