Clobazam Approved as Adjunctive Treatment for Lennox-Gastaut Syndrome

Last week, the FDA announced the approval of Onfi tablets (clobazam) “as an adjunctive (add-on) treatment for seizures” related to Lennox-Gastaut syndrome. With less than 200,000 people diagnosed with Lennox-Gastaut, the FDA classified Onfi as part of its orphan drug designation program, designed for “drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the US”

“Lennox-Gastaut syndrome is a severe form of epilepsy that causes debilitating seizures,” said Russell Katz, MD, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “This is a difficult condition to treat, and it will be helpful to have an additional treatment option.”

Executive VP Anders Gersel Pedersen, Head of R&D at H. Lundbeck A/S said, “Onfi™ has in the largest clinical study ever conducted in LGS patients showed very solid clinical data which was further supported y several key secondary efficacy endpoints.”

Initially prescribed to treat anxiety and convulsions, Onfi was found to be effective in patients two years and older with Lennox-Gastaut through two multicenter controlled studies (CONTAIN Trial), which evaluated seizure control and seizure frequency. Common adverse effects felt by CONTAIN Trial participants included sleepiness, fever, drooling, aggressive behavior, lack of coordination, and constipation; many of which are similar to other anti-epileptic treatments.

Epileptologist Joan A. Conry, MD, professor of neurology at Children’s National Medical Center in DC, and a principal investigator of the CONTAIN Trial, said, “Clobazam, now approved as ONFI, was show to be effective as adjunctive therapy for reducing seizures associated with LGS, and its upcoming availability provides hops for additional seizure management to patients and their physicians, caregivers and families.”