FDA Grants Orphan Drug Designation to Biodel's Glucagon for Prevention of Hypoglycemia in Congenital Hyperinsulinism Population DANBURY, Conn., Dec. 6, 2012 (GLOBE NEWSWIRE) -- Biodel Inc. (Nasdaq:BIOD) today announced that the FDA has granted orphan drug designation for Biodel's 'glucagon' for the prevention of hypoglycemia in the congenital hyperinsulinism (CHI) population. The FDA grants orphan designation to promote development of therapies to treat rare diseases. Once this designation is granted, the sponsor may be eligible for a range of incentives including FDA grant funding for clinical trial costs, tax credits related to development expenses, waiver of FDA user fees, and a seven-year period of marketing exclusivity in the U.S. following FDA approval. Dr.Errol De Souza, President and Chief Executive Officer ofBiodel, stated:"We welcome the FDA's recognition of the important contribution a stable formulation of glucagon could bring to the lives of children suffering from CHI.While our primary glucagon program is a rescue product for the treatment of severe hypoglycemia in patients with diabetes, this designation represents an important component of our strategy to maximize the value of our product candidates to patients and shareholders in both our glucagon and ultra-rapid-acting prandial insulin programs." Biodel previously received a positive opinion for orphan drug designation from the European Medicines Agency's Committee for Orphan Medicinal Products (EMA's COMP) on January 17, 2012, and orphan designation by the European Commission (EC) on March 5, 2012. About Congenital Hyperinsulinism CHI CHI is a genetically heterogeneous disorder characterized by excess, dysregulated insulin secretion from pancreatic beta cells. It is the most common cause of persistent hypoglycemia in neonates and infants and it occurs at an incidence of 1:30,000 to 1:50,000 births. Children who present with this disorder generally require aggressive artificial calorie support in order to prevent hypoglycemia and resulting neurologic damage. Despite aggressive treatment with available therapies, the estimated prevalence of permanent neurologic damage from breakthrough hypoglycemia ranges from 20% to 50%. The treatment paradigm for this condition typically begins with intravenous glucose supplementation, often requiring highly concentrated glucose solutions administered through central venous catheters in intensive care unit settings.While some patients respond to treatment with diazoxide and/or somatostatin analogs, the balance of the patients are eventually treated surgically.Of these, many will need to undergo a near-total pancreatectomy which in most cases leads to insulin-dependent diabetes mellitus by the time the patient reaches adolescence. Glucagon Glucagon is a potential medical treatment for any form of CHI.Glucagon increases blood glucose concentrations acutely by stimulating breakdown of glycogen stores in the liver (glycogenolysis). In the setting of hyperinsulinism, the liver contains excess glycogen due to insulin-dependent inhibition of glycogenolysis.Pilot data describing clinical experience with long-term subcutaneous infusion of glucagon to CHI patients have been published.In a retrospective review of nine patients with CHI, glucagon infusion over weeks to years allowed the reduction or discontinuation of central glucose infusion in all children studied. The glucagon treatment was generally well tolerated, with the most common side effect being a skin rash.However, these glucagon infusions are complicated by frequent catheter obstructions, sometimes occurring daily. This occurs because glucagon in its currently marketed formulations is unstable in solution, particularly at elevated temperatures. This instability in solution makes currently marketed formulations of glucagon impractical for long-term use in these patients. Biodel'sformulation of glucagon is designed to remain stable in solution for a longer period than existing commercial formulations. Preliminary data have been generated to show thatBiodel'sformulation has greater chemical stability at elevated temperatures than existing commercial formulations. In addition,in vitrotesting has shown chemical and physical stability when used in a commercially available insulin pump. AboutBiodel Inc. Biodel Inc.is a specialty biopharmaceutical company focused on the development and commercialization of innovative treatments for diabetes that may be safer, more effective and more convenient for patients. We develop our product candidates by applying our proprietary formulation technologies to existing drugs in order to improve their therapeutic profiles. Safe-Harbor Statement This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements about future activities related to the clinical development plans for the company's drug candidates, including the potential timing, design and outcomes of clinical trials; and the company's ability to develop and commercialize product candidates. Forward-looking statements represent our management's judgment regarding future events. All statements, other than statements of historical facts, including statements regarding our strategy, future operations, future clinical trial results, future financial position, future revenues, projected costs, prospects, plans and objectives of management are forward-looking statements. The words "anticipates," "believes," "could," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. The company's forward-looking statements are subject to a number of known and unknown risks and uncertainties that could cause actual results, performance or achievements to differ materially from those described or implied in the forward-looking statements, including, but not limited to, the success of our product candidates, particularly our proprietary formulations of injectable insulin that are designed to be absorbed more rapidly than the "rapid-acting" mealtime insulin analogs presently used to treat patients with Type 1 and Type 2 diabetes and our liquid formulation of glucagon that is intended to treat patients experiencing severe hypoglycemia; our ability to successfully complete a Phase 2 clinical trial of a proprietary insulin formulation in a timely manner, and the outcome of that trial; our ability to conduct pivotal clinical trials, other tests or analyses required by theU.S. Food and Drug Administration, orFDA, to secure approval to commercialize a proprietary formulation of injectable insulin or a liquid formulation of glucagon; the success of our formulation development work with insulin analog-based formulations of a proprietary injectable insulin and a liquid formulation of glucagon; our ability to secure approval from theFDAfor our product candidates under Section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act; the progress, timing or success of our research, development and clinical programs, including any resulting data analyses; our ability to develop and commercialize a proprietary formulation of injectable insulin that may be associated with less injection site discomfort than Linjeta™ (formerly referred to as VIAject®), which is the subject of a complete response letter we received from theFDA; our ability to enter into collaboration arrangements for the commercialization of our product candidates and the success or failure of any such collaborations into which we enter, or our ability to commercialize our product candidates ourselves; our ability to protect our intellectual property and operate our business without infringing upon the intellectual property rights of others; the degree of clinical utility of our product candidates; the ability of our major suppliers to produce our products in our final dosage form; our commercialization, marketing and manufacturing capabilities and strategies; our ability to accurately estimate anticipated operating losses, future revenues, capital requirements and our needs for additional financing; and other factors identified in our most recent report on Form 10-Q for the quarter endedJune 30, 2012. The company disclaims any obligation to update any forward-looking statements as a result of events occurring after the date of this press release. BIOD-G CONTACT: Seth D. Lewis, +1-646-378-2952
FDA Grants Orphan Drug Designation to Biodel's Glucagon for Prevention of Hypoglycemia in Congenital Hyperinsulinism Population
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