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Home » Xoma’s Congenital Insulin Disorder Drug Wins Orphan Status
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
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