The U.S. Food and Drug Administration has expanded the approval of diazepam nasal spray (Valtoco) for the immediate treatment of seizure clusters in children as young as 2 years old.

Valtoco is a proprietary formulation of diazepam utilizing INTRAVAIL®, a transmucosal absorption enhancement technology that enables rapid intranasal delivery. The formulation was previously granted orphan drug exclusivity after the FDA determined it to be clinically superior to rectal diazepam gel.

The expanded indication is supported by data from a phase I/IIa study evaluating the treatment's safety and pharmacokinetics in pediatric patients; results in patients aged 2 years and older were presented at the 2024 meetings of the Child Neurology Society and the American Epilepsy Society. The most common adverse events reported were somnolence, headache, and nasal discomfort.

“Valtoco fills a large unmet need for children with seizures and their families. I am hopeful that this product will improve quality of life for this specific population," said Eric Segal, MD, of Northeast Regional Epilepsy Group and Hackensack University Medical Center.

Seizure clusters, or acute repetitive seizures, are intermittent episodes that differ from a patient’s usual seizure pattern. While many patients achieve control of chronic epilepsy with medication, some continue to experience episodes of frequent seizures. Approximately 400,000 children in the United States are living with epilepsy.

“Approval of an immediate-use intranasal diazepam treatment that can be given at any time—even while a child is actively seizing—in this young age group is an important advance for the epilepsy community," said Jurriaan M. Peters, MD, PhD, of Boston Children’s Hospital and Harvard Medical School.

Valtoco carries a boxed warning regarding the risks of concomitant opioid use, abuse and misuse, physical dependence, and withdrawal reactions. It is contraindicated in patients with hypersensitivity to diazepam, acute narrow-angle glaucoma, or central nervous system depression. Valtoco is not approved for use in neonates or infants due to the risk of benzyl alcohol–related toxicity.

Valtoco is classified as a Schedule IV controlled substance. Full prescribing information, including boxed warning, is available at www.valtoco.com.

Reference:

Neurelis Announces FDA Approval for Immediate Use Seizure Medication VALTOCO® (Diazepam Nasal Spray) in Ages 2 to 5. [press release]