FDA approves Nayzilam for epilepsy

UCB announced that the FDA has approved a New Drug Application for the company's newest anti-epileptic drug (AED) Nayzilam (midazolam) nasal spray CIV, a benzodiazepine indicated for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient's usual seizure pattern in patients with epilepsy 12 years of age and older. Nayzilam now provides patients and caregivers with the first and only FDA-approved nasal option for treating seizure clusters.

The effectiveness of Nayzilam for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient's usual seizure pattern in patients with epilepsy 12 years of age and older was established in a randomized, double-blind, placebo-controlled trial (Study 1; NCT 01390220). Study 1 enrolled patients with epilepsy on a stable regimen of antiepileptic drugs who were identified by their physicians as having intermittent, stereotypic episodes of frequent seizure activity that were distinct from the patient's usual seizure pattern. Study 1 was conducted in two phases: an open-label Test Dose Phase followed by a randomized, double-blind, placebo-controlled, Comparative Phase. In the Comparative Phase, 201 patients treated a single seizure cluster episode in an outpatient setting with either a blinded dose of Nayzilam 5 mg (134 patients) or placebo (67 patients). A statistically significantly higher percentage of Nayzilam-treated patients met the primary efficacy endpoint. Numerical differences in favor of Nayzilam were observed on each of the components of the treatment success responder definition; termination of seizure(s) within 10 minutes after initial dose of study drug (80.6 versus 70.1%) and the absence of seizure recurrence between 10 minutes and 6 hours after the initial dose of study drug (58.2 versus 37.3%). The most common adverse reactions (at least5% in any Nayzilam treatment group) were somnolence, headache, nasal discomfort, throat irritation, and rhinorrhea.