Insmed Receives FDA Orphan Drug Designation For rhIGF-I/rhIGFBP-3, SomatoKine, for Extreme Insulin Resistance.
Business Editors/Health/Medical Writers
RICHMOND, Va.--(BUSINESS WIRE)--Dec. 15, 2003
Insmed Incorporated (Nasdaq: INSM INSM Initiative Neue Soziale Marktwirtschaft (German)
INSM Integrated Network and Systems Management ) today announced that the Office of Orphan Products Development of the Food and Drug Administration has approved the Company's application for orphan drug orphan drug, drug developed under the U.S. Orphan Drug Act (1983) to treat a disease that affects fewer than 200,000 people in the United States. The orphan drug law offers tax breaks and a seven-year monopoly on drug sales to induce companies to undertake the designation in the United States for SomatoKine(R), for the treatment of extreme insulin resistance. With this orphan drug designation, Insmed will be granted seven years of market exclusivity upon SomatoKine(R) approval for this indication. There is currently no approved treatment available for patients with extreme insulin resistance in the United States.
Insmed's investigational drug rhIGF-I/rhIGFBP-3, SomatoKine(R), is a proprietary delivery composition of insulin like growth factor-I (IGF-I IGF-I
see somatomedin C.
IGF-I Insulin-like growth factor I, somatomedin-C A polypeptide hormone structurally similar to proinsulin, synthesized in the liver and fibroblasts, giving fibroblasts a paracrine function; serum levels correlate with ). 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 anabolic
pertaining to or arising from anabolism.
steroids with a tissue-building effect. Testosterone is an example of a natural anabolic steroid with the, sometimes undesirable, effect of causing masculinization. disorders where IGF (Internet Governance Forum) An international organization of governments and U.N. agencies that was founded to discuss Internet issues such as security and spam. It was created at the United Nations Summit in 2005 after the U.S. deficiency exists. Insmed is currently developing rhIGF-I/rhIGFBP-3 for the treatment of severe growth disorders and diabetes.
More on Extreme Insulin Resistance
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 Hirsutism Definition
Excessive growth of facial or body hair in women is called hirsutism.
Hirsutism is not a disease. The condition usually develops during puberty and becomes more pronounced as the years go by. , and dysmorphic features.
More on rhIGF-I/rhIGFBP-3, SomatoKine(R)
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 bone mineral density
See bone density.
bone mineral density A measurement of bone mass, expressed as the amount of mineral–in grams divided by the area scanned in cm2. See Bone densitometry. . rhIGF-I/rhIGFBP-3 is currently in a pivotal Phase III clinical trial Noun 1. phase III clinical trial - a large clinical trial of a treatment or drug that in phase I and phase II has been shown to be efficacious with tolerable side effects; after successful conclusion of these clinical trials it will receive formal approval from the for the treatment of Growth Hormone Insensitivity Syndrome growth hormone insensitivity syndrome Laron dwarfism, pituitary dwarfism II An AR condition characterized by severe growth retardation, delayed bone age, occasionally blue sclerae, and physical manifestations of GH deficiency, due to a defect in the GH receptor (GHIS GHIS Growth Hormone Insensitivity Syndrome
GHIS Geosynchronous High-Resolution Interferometer Sounder ), a severe growth disorder.
About Insmed Incorporated
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. For more information, please visit www.insmed.com.
Statements included within this press release, which are not historical in nature, may constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act The Private Securities Litigation Reform Act of 1995 (PSLRA) implemented several significant substantive changes affecting certain cases brought under the federal securities laws, including changes related to pleading, discovery, liability, class representation and awards fees and of 1995. Forward-looking statements include all statements regarding expected financial position, results of operations, cash flows, dividends, financing plans, business strategies, operating efficiencies or synergies, budgets, capital and other expenditures, competitive positions, growth opportunities for existing or proposed products or services, plans and objectives of management, demand for new pharmaceutical products, market trends in the pharmaceutical business, inflation and various economic and business trends. Such forward-looking statements are subject to numerous risks and uncertainties, including risks that product candidates may fail in the clinic or may not be successfully marketed, the company may lack financial resources to complete development of product candidates, competing products may be more successful, demand for new pharmaceutical products may decrease, the biopharmaceutical industry may experience negative market trends and other risks detailed from time to time in the company's filings with the Securities and Exchange Commission. As a result of these and other risks and uncertainties, actual results may differ materially from those described in this press release.