Belgian pharmaceutical company UCB recently announced that the US FDA has approved its latest antiepileptic drug (AED) Nayzilam (midazolam) nasal spray CIV. This is a benzodiazepine drug for nasal administration. It is suitable for the emergency treatment of intermittent and stereotyped seizures (such as epileptic cluster seizures, acute recurrent seizures) for patients with epilepsy 12 years old and above.

Nayzilam is the first and only FDA-approved treatment for epilepsy clusters through nasal administration, and the first new drug approved for the treatment of epilepsy clusters in the US market for more than 20 years. Its nasal spray administration method has limited options for current treatments. Patients and their caregivers offer a new solution.

It is estimated that in the United States, more than 150,000 patients with uncontrolled epilepsy (UCE) will also experience seizure clusters, and rescue treatment is essential because, if left untreated, seizure clusters will increase physical injury, nerve damage, Risk of persistent seizures and status epilepticus (SE). Although affected by seizure clusters, many confirmed patients may not be treated because the currently available treatment options (rectal benzodiazepines such as diazepam [diazepam, diazepam]) are not desirable.

Nayzilam is a short-acting drug that treats epileptic clustered seizures through nasal sprays. The drug is intended as a one-time therapy that is convenient for patients to carry and can be administered by a non-medical professional when a seizure occurs in a patient.

This approval is based on data from the Phase III clinical study ARTEMIS-1 (NCT01390220). This is a randomized, double-blind, placebo-controlled study evaluating the use of Nayzilam in the treatment of epilepsy clusters in patients with epilepsy. The study was divided into two phases: the open-label trial dose phase, followed by a randomized, double-blind, placebo-controlled comparison phase. During the trial dose phase, tolerability assessments were performed on 292 patients who received two 5 mg doses of Nayzilam without seizures at 10-minute intervals. Patients were excluded from the comparison phase if they failed to meet predefined blood pressure, heart rate, sedation, electrocardiogram, and peripheral oxygen saturation criteria.

During the comparison phase, 201 patients were blinded to Nayzilam 5mg (n = 134) or placebo (n = 67) for a single epileptic cluster in the outpatient setting. If epileptic activity persists or relapses, both groups of patients can choose to receive Nayzilam 5mg non-blind treatment within 10 minutes to 6 hours after the initial blind treatment. The study's primary efficacy endpoint was treatment success, which was defined as stopping seizures within 10 minutes of initial blind drug administration and no recurrence of epilepsy within 6 hours after initial blind drug administration.

The results showed that the proportion of patients who reached the primary endpoint in the Nayzilam treatment group was statistically significantly higher, and a numerical value in favor of Nayzilam was observed for each component defined by the successful treatment: stop epilepsy within 10 minutes after the initial blind administration Seizures (80.6% vs 70.1%), seizures did not recur within 10 minutes to 6 hours (58.2% vs 37.3%). The most common adverse reactions in the study were drowsiness, headache, nasal discomfort, throat irritation, and runny nose.

The study also evaluated the next seizure rate and duration after the initial blind administration of study drug. The results showed that compared with the placebo group, the Nayzilam treatment group had a lower proportion of patients with recurrent seizures within 24 hours after the initial blind drug administration (37.3% vs 46.3%). Statistically longer.

Nayzilam was acquired by Ushibee after acquiring Proximagen for $ 370 million in June 2018. The acquisition aims to expand its epilepsy drug pipeline. Earlier, the FDA had granted Nayzilam fast track status and orphan drug status.