Insmed Incorporated has submitted a New Drug Application (NDA) to the US FDA for regulatory approval of SomatoKine (mecasermin rinfabate) for the treatment of growth hormone insensitivity syndrome (GHIS).

The application includes safety and efficacy results from Insmed's prospective, multi-centre, phase III clinical trial with SomatoKine conducted in patients with growth hormone insensitivity syndrome. SomatoKine is Insmed's proprietary insulin-like growth factor I (IGF-I) therapy, composed of recombinant human IGF-I and IGF binding protein 3 (rhIGF-I/rhIGFBP-3 complex).

"The submission of the NDA for SomatoKine represents a great achievement for the Company, highlighting our efforts to bring the first specific treatment for children with this disease. We believe that SomatoKine is a breakthrough therapeutic agent and that it will become the standard of care for treating GHIS," said Geoffrey Allan, president and CEO of Insmed Incorporated.

SomatoKine has already received orphan drug designation for the GHIS indication. The GHIS indication encompasses a variety of genetic and acquired conditions in which the action of growth hormone (GH) is absent or severely attenuated, resulting in low serum levels of IGF-I. Because IGF-I is the primary mediator of the growth-promoting actions of GH, replacement therapy in children with GHIS using SomatoKine is intended to bypass the blockade of GH action and improve longitudinal growth, the company said.

Insmed is a biopharmaceutical company focused on the discovery and development of drug candidates for the treatment of metabolic diseases and endocrine disorders with unmet medical needs.