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Drug intelligence / Profile preview
Onfi
- Development stage
- Approved
- Lead developer
- Lundbeck
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Onfi (clobazam) is a small molecule benzodiazepine anticonvulsant developed by H. Lundbeck A/S. It acts as a positive allosteric modulator of the gamma-aminobutyric acid type A (GABA-A) receptor. By binding to these receptors, clobazam enhances the inhibitory effect of GABA, increasing chloride ion conductance and stabilizing hyperexcitable neuronal membranes to prevent seizures. Onfi is primarily approved in the United States as an adjunctive therapy for seizures associated with Lennox-Gastaut Syndrome (LGS). While it is frequently used off-label for the management of Dravet syndrome, Lundbeck's specific clinical development program for this indication (including trials NCT02174094 and NCT02187809) was withdrawn in 2015 due to recruitment difficulties.
- Brand names
- Onfi
- Other names
- clobazam
02
Targets
BZD site (GABA-A receptor benzodiazepine site)δ-GABAAR (Extrasynaptic GABA-A receptor delta-containing subtype)α6β2δ GABAAR (GABA-A receptor α6β2δ subtype)GABAA (Gamma-aminobutyric acid receptor)
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