pregnanolone pyroglutamate

Pregnanolone pyroglutamate is a synthetic prodrug of the endogenous neurosteroid pregnanolone (3α,5β-P), specifically designed to treat pediatric epilepsy and pharmacoresistant seizures. The molecule features a pyroglutamate moiety at the C-3 position, which masks the metabolically labile hydroxyl group to improve drug-like properties and facilitate delivery. Upon administration, the prodrug is enzymatically cleaved in vivo to release the parent neurosteroid. Pregnanolone then acts as a potent positive allosteric modulator of γ-aminobutyric acid type A (GABA_A) receptors, enhancing inhibitory neurotransmission in the central nervous system. Preclinical studies in rat models of the developing brain have demonstrated that this prodrug strategy significantly reduces seizure duration and severity, showing superior stability and efficacy compared to unmodified neurosteroids.

Other names

3α-hydroxy-5β-pregnan-20-one pyroglutamatepregnanolone-3-pyroglutamatepregnanolone3-pyroglutamatepregnanolone 3-pyroglutamate