Investigational Therapy Designed to Stop Epileptic Seizures
Staccato Alprazolam is a hand-held inhaler in development that rapidly terminates seizure activity once it has begun. The investigational therapy combines the easy-to-use, FDA-approved Staccato delivery technology with alprazolam, a well-known benzodiazepine that has potent anti-epileptic properties.
How Staccato Alprazolam is Designed to Work
A small inhaler that delivers alprazolam with a single, natural breath, Staccato Alprazolam is being studied as a way for people with epilepsy and their caregivers to potentially stop an ongoing seizure.
The Staccato system is a small, portable device that consists of three components: a heating substrate, a thin film of the drug coated on the substrate, and an airway through which the patient inhales. After removing its packaging, the patient must simply place the device to his or her lips and take a breath. The Staccato system rapidly vaporizes the drug to form a small particle aerosol, which is drawn through the patient’s mouth and into the lungs, where it diffuses into circulation where it is designed to have a rapid systemic effect.
A Streamlined Path to FDA Approval
While there are other types of epileptic seizure rescue therapies on the market, there are no FDA-approved options that offer the practicality of an inhaler. Engage Therapeutics is using the 505(b)(2) regulatory pathway to develop and seek FDA approval for Staccato Alprazolam, an abbreviated route to approval offered to new products that rely, in part, on earlier-developed clinical data since alprazolam has previously been approved by the FDA for other uses.
In a phase 2a study, Staccato Alprazolam demonstrated a reduction in seizure-like activity in patients with photosensitive seizures. To move forward in the approval process, Engage Therapeutics is recruiting participants for a phase 2b study called StATES (Staccato Alprazolam Terminates Epilepsy Seizures). The clinical trial aims to test the efficacy of Staccato Alprazolam in naturally-occurring seizures. Should the study obtain positive results, there will be one final trial prior to requesting FDA approval and market release of the product.
The Future of Seizure Rescue Therapy
Epilepsy affects at least three million people in the United States. A third of these people suffer from refractory epilepsy with uncontrolled seizures, for which anti-epileptic drugs do not help. We’re developing Staccato Alprazolam to stop seizures in people with predictable seizure patterns.
Technology to predict oncoming seizures is on the horizon, but awareness of an impending seizure is useless without a plan. Coupling these technologies in the future could allow us to stop seizures before they even begin—limiting the physical and psychological burden that seizures cause.