Xoma’s Congenital Insulin Disorder Drug Wins Orphan Status

June 22, 2015

The FDA has granted orphan drug designation to Berkeley, Calif., drugmaker Xoma’s investigational treatment for congenital hyperinsulinism, a life-threatening disease that causes the body to produce excessive amounts of insulin.

Roughly 1 in 50,000 newborns have HI, which forces blood sugar levels to plunge dangerously, possibly leading to brain damage or death. Current treatment options include continuous ingestion of glucose or a pancreatectomy, the company says.

In a recent Phase 1 study, healthy volunteers who received Xoma 358 saw their insulin receptor signaling drop while glucose production increased. The drug was also well-tolerated with no serious adverse events observed. 

The FDA grants orphan drug designation to novel drugs or biologics that treat a rare disease affecting fewer than 200,000 patients in the U.S. The designation comes with a seven-year period of U.S. marketing exclusivity, assistance with clinical trial design and waiver of PDUFA filing fees. — John Bechtel