Onfi (clobazam) is an anticonvulsant therapy marketed by Lundbeck. It is approved by the U.S. Food and Drug Administration (FDA) as an add-on therapy to treat Lennox-Gastaut Syndrome (LGS), an epilepsy disorder affecting children.

Onfi can also be used to reduce the severity of seizures in children with Dravet syndrome, usually in combination with another anticonvulsant such as Depacon (sodium valproate).

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to investigate the efficacy and long-term safety of Onfi as an add-odd therapy in children with Dravet syndrome — were withdrawn in 2015 due to recruitment difficulties.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.

Other information

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.

***

Dravet Syndrome News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas. Emily is passionate about science communication, and, in her free time, writes and illustrates children’s stories.
×
Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas. Emily is passionate about science communication, and, in her free time, writes and illustrates children’s stories.
Latest Posts
    The User does not have any posts