LJPC-0712

LJPC-0712 is an investigational **allopregnanolone** program developed by **La Jolla Pharmaceutical Company** for **Niemann-Pick type C disease**. Allopregnanolone is an endogenous neurosteroid and a progesterone metabolite that acts as a **positive allosteric modulator of gamma-aminobutyric acid type A receptors**, enhancing GABAergic signaling in the central nervous system. In this program, it is being developed as a pharmaceutical neurosteroid for a rare inherited lysosomal storage disorder caused by mutations in **NPC1** or **NPC2**, and it has been reported to have received **Orphan Drug designation** from the FDA for Niemann-Pick type C disease.

Other names

allopregnanolone