FDA approval for Valtoco (diazepam nasal spray) for short-term treatment of seizure clusters (also known as “acute repetitive seizures”).- Neurelis Inc.

Neurelis, Inc., announced that the FDA has approved Valtoco (diazepam nasal spray) for short-term treatment of seizure clusters (also known as “acute repetitive seizures”) that are different from a person’s normal seizure pattern in people 2 years of age and older.  Valtoco is a proprietary formulation which utilizes an absorption enhancement technology, INTRAVAIL, to enable the noninvasive, enhanced intranasal delivery of diazepam.  The intranasal formulation of Neurelis’ Valtoco was previously recognized by the FDA as clinically superior to the rectal gel formulation of diazepam resulting in its orphan drug exclusivity designation.  

“The FDA’s decision to approve Valtoco for use in early childhood highlights the established balanced safety and efficacy profile,” commented Eric Segal, MD, Director of Pediatric Epilepsy at Northeast Regional Epilepsy Group and Hackensack University Medical Center. “ 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.” 

Approximately 3.4 million people in the U.S. have epilepsy, including 400,000 children.  While chronic epilepsy can be controlled by medications, some patients are at risk of episodes of frequent seizures, or acute repetitive seizures.

“For children who have episodes of frequent seizures, the current standard of care requiring rectal administration of medication to stop a seizure can be a significant challenge for caregivers and children alike,” added Jurriaan M Peters, MD, PhD, Director, Localization Laboratory, Division of Epilepsy and Clinical Neurophysiology, Boston Children’s Hospital, and Associate Professor in Neurology at Harvard Medical School.  “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.”

Data from the phase 1/IIa clinical study evaluating safety and pharmacokinetics demonstrated that Valtoco  is safe and effective with easy nasal administration for patients aged 2 years and older who have episodes of frequent seizures.  The company presented data on the study at the 53rd Annual Child Neurology Society Meeting in November 2024 in San Diego and Annual American Epilepsy Society Meeting in December 2024 in Los Angeles.