The FDA has granted orphan drug status to Biodel’s stable glucagon for congenital hyperinsulinism patients. This represents one more important milestone in the development of this drug for HI patients. This FDA designation creates a range of financial incentives for the further development of the drug. Earlier this year, Biodel had received orphan drug designation or its equivalent from the European Commission.
The form of glucagon that Biodel is developing is a breakthrough for the HI population because it is a more stable form of the drug. The medication that is currently on the market is unstable and therefore problematic. The new, more stable form of the drug could be useful to HI patients in three distinct ways.
Glucagon is used by HI patients as an emergency medication when severe hypoglycemia occurs. Currently, patients or their parents must pre-mix and draw up the medication into a syringe. Obviously, this is far from ideal when time is of the essence and the glucagon must be delivered as fast as possible to prevent prolonged severe hypoglycemia which can cause brain damage. The new form of glucagon will allow patients or their parents to inject a ready-to-use product.
The new glucagon may also be more suitable for long-term therapy for HI patients when other alternative treatments are not effective. The medication could possibly be delivered on this basis subcutaneously using an insulin pump or something like it.
In another important development, this stable glucagon could also be a key component for use in a bi-hormonal pump for the treatment of diabetes in HI patients who have become diabetic after sub-total pancreatectomies. While the bi-hormonal pumps would be a great development for all diabetes patients, they represent something particularly important for HI diabetes patients. Some HI patients with diabetes still produce some insulin at unexpected times in an unregulated manner. These patients must deal with and worry about lows as well as highs perhaps to an even greater extent than the type 1 diabetes population. A back-up system in which glucagon is available and can be delivered when blood sugar levels dip too low is a fantastic development. Preclinical trials studying the use of the new glucagon in bi-hormonal pumps are currently underway.
You can read more about Biodel’s news here: http://files.shareholder.com/downloads/BIOD/2206854174x0x620550/ecce3afc-517f-4fba-9c14-5a61bad504cf/BIOD_News_2012_12_6_General_Releases.pdf
