Insmed Incorporated announced that the Office of Orphan Products Development of the FDA has approved the company's application for orphan drug designation in the US for SomatoKine, for the treatment of extreme insulin resistance. With this orphan drug designation, Insmed will be granted seven years of market exclusivity upon SomatoKine approval for this indication. There is currently no approved treatment available for patients with extreme insulin resistance in the US.
Insmed's investigational drug rhIGF-I/rhIGFBP-3, SomatoKine, is a proprietary delivery composition of insulin like growth factor-I (IGF-I). This drug is administered as a once-daily subcutaneous injection and is designed to restore IGF-I levels to more normal ranges in metabolic and anabolic disorders where IGF deficiency exists. Insmed is currently developing rhIGF-I/rhIGFBP-3 for the treatment of severe growth disorders and diabetes.
Syndromes of extreme insulin resistance appear to result from genetic defects in the insulin receptor or insulin action pathways. In addition to insulin resistance and glucose intolerance or overt diabetes, these syndromes share a number of common features including variable degrees of hyperandrogenism, hirsutism, and dysmorphic features.
Insmed's rhIGF-I/rhIGFBP-3 is a proprietary delivery composition of insulin-like growth factor-I (IGF-I). The novel compound is administered as a once-daily subcutaneous injection, which can restore IGF levels to physiological relevant levels. In diabetic subjects, administration of rhIGF-I/rhIGFBP-3 demonstrated a significant improvement in blood sugar control and a significant reduction in daily insulin use. Following severe burn injury, in both children and adults, administration of rhIGF-I/rhIGFBP-3 demonstrated a significant improvement in muscle protein synthesis and a significant reduction in the inflammatory response associated with the trauma. In recovery from hip fractures, administration of rhIGF-I/rhIGFBP-3 has demonstrated a significant improvement in functional recovery and bone mineral density. rhIGF-I/rhIGFBP-3 is currently in a pivotal Phase III clinical trial for the treatment of Growth Hormone Insensitivity Syndrome (GHIS), a severe growth disorder.