FDA Approves Neurelis' Valtoco Nasal Spray for Ages 2-5

Neurelis, Inc. has announced that the U.S. Food and Drug Administration (FDA) has approved VALTOCO® (diazepam nasal spray) for the short-term treatment of seizure clusters in individuals aged two years and older. This formulation is designed to address episodes of frequent seizures that differ from an individual's typical seizure pattern. VALTOCO utilizes INTRAVAIL®, an absorption enhancement technology, allowing for effective intranasal delivery of diazepam.

The FDA previously recognized VALTOCO's intranasal formulation as superior to the rectal gel form of diazepam, granting it orphan drug exclusivity. Neurelis Founder and CEO, Craig Chambliss, expressed gratitude to the patients and families involved in the clinical trials that facilitated this approval, highlighting the importance of providing an immediate-use medication to manage frequent seizure episodes.

Eric Segal, MD, Director of Pediatric Epilepsy at Northeast Regional Epilepsy Group, emphasized the importance of this approval for young children, noting the product's balanced safety and efficacy profile. He believes that VALTOCO meets a significant unmet need for children with seizures and their families, potentially improving their quality of life.

Seizures are a common challenge in epilepsy, affecting about 3.4 million people in the United States, including 400,000 children. While medications often control chronic epilepsy, some patients are prone to episodes of frequent seizures. Jurriaan M Peters, MD, PhD, from Boston Children's Hospital, pointed out that the current standard treatment, which often involves rectal medication administration, presents difficulties for both caregivers and children. The new approval of an intranasal diazepam treatment allows for administration even during a seizure, marking a significant advancement for the epilepsy community.

Clinical studies, including a phase 1/2a trial, have demonstrated the safety and efficacy of VALTOCO for patients aged two and older. These studies were presented at the Child Neurology Society and American Epilepsy Society meetings in 2024.

Neurelis, Inc., a company focused on neuroscience, aims to develop and commercialize therapeutics for epilepsy and related neurological disorders with high unmet needs. Besides VALTOCO, Neurelis is also working on NRL-1004, a Phase 1 intranasal olanzapine for treating acute agitation in conditions like schizophrenia and bipolar disorder. Another development is NRL-1049, a Phase 1 Rho kinase inhibitor for cerebral cavernous malformations, a central nervous system disorder.

Important safety information for VALTOCO includes its indication for acute treatment of specific seizure episodes in patients aged two and older. The nasal spray is contraindicated for individuals with hypersensitivity to diazepam, acute narrow-angle glaucoma, and central nervous system depression. Benzodiazepines like VALTOCO can cause CNS depression, so patients should avoid activities requiring mental alertness until the drug's effects diminish. There's also a risk of increased suicidal ideation associated with antiepileptic drugs, emphasizing the need for monitoring any mood changes.

VALTOCO is not approved for neonates or infants due to the risk of serious adverse reactions, including "gasping syndrome" caused by benzyl alcohol preservative. Common side effects include somnolence, headache, and nasal discomfort. Diazepam, the active component of VALTOCO, is classified as a Schedule IV controlled substance.