Great news for the epilepsy community! The FDA has just approved a new diazepam nasal spray (Valtoco; Neurelis Inc), specifically designed for the short-term treatment of seizure clusters, also known as acute repetitive seizures, in people 2 years and older. This breakthrough could make a real difference for those experiencing this challenging condition -- especially pediatric patients.
This approval follows the earlier orphan drug designation for the diazepam nasal spray, which uses Intravail (Aegis Therapeutics). This technology enhances the noninvasive absorption of Diazepam through the nasal mucosa.
But first... What are seizure clusters?
Seizure clusters happen when someone has multiple seizures in a short amount of time—think of it as a rough patch that can feel overwhelming. For many folks with epilepsy, these clusters can be scary and might require urgent medical attention. Until now, the options for handling these situations were often complicated—administering rescue medications generally involved more invasive methods like intravenous or rectal delivery, which can be tough for both patients and their caregivers.
Now, Diazepam is a name you might recognize—it's been around for quite some time and belongs to a class of medications called benzodiazepines. It's well-known for its ability to reduce anxiety, relax muscles, and, importantly, act as an anticonvulsant. But now, with the new nasal spray formulation, it's set to change the game when it comes to treating seizure clusters.
The approval of the Diazepam nasal spray was supported by the phase 1/2a Stellina clinical study. This study evaluated a single dose of diazepam as a rescue medication for children aged 2 to 5 with epilepsy. It assessed the pharmacokinetics of the nasal spray and monitored long-term safety over six months.
Thirty-five pediatric patients participated, with an average age of 3.9 years. Doses were adjusted based on body weight and given by caregivers for immediate seizure cluster treatment, totaling 299 doses across three levels: 5 mg (11 doses), 10 mg (238 doses), and 15 mg (50 doses). Safety was monitored at 30, 90, and 180 days.
Of the participants who completed monitoring, 20% reported mild treatment-related side effects, such as drowsiness, headaches, and nasal discomfort. The safety profile in children matched that of older patients, confirming the treatment's suitability for this age group.
With this new nasal spray in the toolbox, healthcare providers are gaining a valuable addition to their arsenal for managing epilepsy. It empowers both patients and caregivers, making them feel more prepared to face seizure clusters—whether at home, school, or out in the community. The approval of this nasal spray represents a significant step toward enhancing the quality of life for those with epilepsy, aiming to minimize risks associated with seizure episodes and improve overall health outcomes.
Sources:
https://pmc.ncbi.nlm.nih.gov/articles/PMC8366630/
https://pubmed.ncbi.nlm.nih.gov/16822227/
https://www.ncbi.nlm.nih.gov/sites/books/NBK470159/
