Onfi (clobazam) is an anticonvulsant therapy marketed by Lundbeck. It is approved by the U.S. Food and Drug Administration as an add-on therapy to treat Lennox-Gastaut syndrome, an epilepsy disorder affecting children.
How Onfi works
Nerve signals sent from the brain travel along nerve cells (neurons) to muscle cells, controlling their movement. Such signals are sent by changing the flow of charged particles in and out of nerve cells. After a signal is sent, the nerve cell must be “reset” to make way for a new signal.
In Dravet syndrome, mutations in a protein involved in the transport of charged particles in and out of nerve cells cause overactive nerve signaling, or misfires, that result in muscle tremors and seizures.
Onfi belongs to a class of chemicals called benzodiazepines, and works by binding to a receptor protein found in neurons called GABA. GABA is part of a signaling complex in neurons, which inhibits nerve signaling once a signal has been sent, preventing tremors or muscle spasms following movement.
When bound to GABA receptors, Onfi increases the flow of charged particles into the neuron, preventing overactive nerve signaling and subsequent seizure activity.
Onfi in clinical trials for Dravet
Two Phase 3 clinical trials planned by Lundbeck — (NCT02174094 and NCT02187809) — to investigate the efficacy and long-term safety of Onfi as an add-odd therapy in children with Dravet syndrome — were withdrawn in 2015 due to recruitment difficulties.
A single retrospective study published in the scientific journal Epilepsy Research compared the effect of the ketogenic diet and other therapies, including Onfi, on seizure frequency by type in 32 children with Dravet syndrome. The response rate for the combination of Decapon, Onfi, and Diacomit was 89 percent, while the response rate for Onfi alone was 28 percent.
Onfi is associated with side effects that include drowsiness or dizziness, slurred speech, loss of balance or coordination, and drooling. Sleep problems, nausea or vomiting have also been reported.
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