La Jolla Gets Orphan Drug Status For LJPC-0712 - Quick Facts

(RTTNews) - La Jolla Pharmaceutical Co. (LJPC.OB), a developer of therapeutics targeting life threating diseases, Wednesday said its drug candidate LJPC-0712 was granted an Orphan Drug designation by the FDA Office of Orphan Products Development. LJPC-0712, commonly known as allopregnanolone, is used in the treatment of Niemann-Pick type C disease.

Niemann-Pick type C disease (NP-C) is a rare inherited disease caused by mutations in either the NPC1 or of the NPC2 gene. These mutations lead to impaired intracellular lipid trafficking with subsequent accumulation of cholesterol and glycosphingolipids in the brain and other tissues.

Allopregnanolone is a neurosteroid present in the blood and brain. It is a metabolite of progesterone and potent modulator of gamma-aminobutyric acid or GABAA receptors. It increases the activity of endogenous GABA by allosteric modification of the GABAA receptor. Allopregnanolone has pharmacological properties similar to other positive modulators of GABAA receptors, including anti-anxiety and anti-seizure activity.