FDA grants orphan drug designation to Biodel drug for rare insulin disease

FDA grants orphan drug designation to Biodel drug for rare insulin disease
Biodel receives designation for glucagon to treat congenital hyperinsulinism


December 6, 2012 | By Alaric DeArment

DANBURY, Conn. — The Food and Drug Administration has given special designation to a treatment made by Biodel for patients with congenital hyperinsulinism, a rare disorder that affects children.

Biodel announced that it had received orphan drug designation for glucagon to prevent hypoglycemia, or abnormally low blood sugar, in patients with CHI.

The FDA grants orphan drug designation to treatments for rare diseases. The designation makes the companies receiving it eligible for incentives like FDA grant-funding for clinical trial costs, tax credits, waiver of user fees and a seven-year period of marketing exclusivity following regulatory approval, compared with the five that new drugs normally receive.

CHI occurs in 1-in-30,000 to 50,000 births, and children who have it generally need aggressive artificial calorie support to prevent hypoglycemia and neurologic damage, but neurologic damage still occurs in about 20% to 50% of cases.