NORD Honors Insmed for Role in Developing Treatments for Rare Diseases
--------------------------------------------------------------------------------
PRNewswire
09:31 a.m. 04/25/2006
Company Saluted for Research in Treating Children With Extreme Short Stature
RICHMOND, Va., April 25, 2006 /PRNewswire-FirstCall via COMTEX/ -- Insmed Incorporated (INSM) was honored by the National Organization for Rare Disorders (NORD) last night at its annual Tribute Banquet for the development of IPLEX(TM) (mecasermin rinfabate (rDNA origin) injection) for the treatment of growth failure in children with severe primary IGF-1 deficiency (Primary IGFD) or with growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH. IPLEX was approved by the United States Food and Drug Administration in December 2005 as an orphan drug.
"For many years we've recognized the need for treatment for Primary IGF-1 deficiency, but few companies were willing to commercially develop insulin- like growth factor until now. IPLEX(TM) is a significant medical advancement worthy of recognition and we thank Insmed for this important contribution," said Abbey S. Meyers, President of NORD.
"We applaud NORD's longstanding tradition of recognizing those that dedicate their life's work to finding treatments for rare diseases, and we are delighted to be included in this celebration," said Ronald D. Gunn, M.B.A., Executive Vice President and Chief Operating Officer of Insmed. "IPLEX(TM) is the culmination of over 20 years of dedicated research and development, and represents a breakthrough in IGF-1 replacement therapy, with potential benefits in several additional rare diseases with unmet medical needs."
About IPLEX
IPLEX, a complex of rhIGF-1 and rhIGFBP-3, is the only once-daily IGF-1 replacement therapy and the only therapy that provides both IGF-1 and IGFBP-3 that is approved by the FDA to treat children with severe Primary IGFD. The drug, expected to launch during the second quarter of calendar year 2006, is also being studied for various other indications with unmet medical needs including extreme insulin resistance, myotonic muscular dystrophy and HIV Associated Adipose Redistribution Syndrome (HARS).
About the Condition
Severe Primary IGFD is a genetic condition in which patients do not generate sufficient quantities of insulin-like growth factor-I (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3), two key proteins involved in mediating the effects of growth hormone. Patients with severe Primary IGFD present with extreme short stature (height standard deviation score, SDS < -3) and an extremely poor prognosis for adult stature. Whereas prepubertal height SDS can range as low as -9, a blunted pubertal growth spurt is usually apparent, resulting in adult height that is typically 5-12 SD's below the normal population (adult height between 100 and 140 cm). The profound short stature in patients is often associated with severe psychosocial problems.
