FDA Accepts New Drug Application for Nayzilam Nasal Spray for Acute Treatment of Seizures

The U.S. Food and Drug Administration has accepted a new drug application (NDA) for UCB’s Nayzilam (midazolam) nasal spray for the acute treatment of seizures including seizure clusters and acute repetitive seizures.

Acceptance of this NDA could result in Nayzilam’s approval in the U.S. in early 2019, potentially making it the first new medication approved to treat seizure clusters in more than 17 years.

Nayzilam nasal spray is an investigational rescue medication intended to treat prolonged seizures, which can occur in Batten disease patients.

Most seizures are brief, but a single seizure lasting longer than five minutes, or a cluster of seizures during which the patient does not regain consciousness, called status epilepticus, can be fatal and constitutes a medical emergency. Rescue medications, given to stop a prolonged seizure in progress, can be crucial in these cases.

“Managing seizure clusters remains a challenge for thousands of patients and caregivers, in the US and beyond, who live their lives each day with this debilitating condition,” said Jeff Wren, head of neurology and executive vice president of UCB, in a press release.

UCB’s NDA was supported by data from a Phase 3 clinical trial, called ARTEMIS-1 (NCT01390220), which evaluated the safety and effectiveness of Nayzilam in 292 patients who experienced episodes of acute repetitive seizures.

ARTEMIS-1 consisted of two phases: an in-clinic test dose phase and an outpatient comparative phase. During the test dose phase, patients with a history of seizure clusters receiving a stable regimen of anti-epileptic medications were given two 5 mg doses of open-label Nayzilam 10 minutes apart at the study center. In the comparative phase, subjects were randomized to receive 5 mg of Nayzilam or a placebo.

Caregivers administered the double-blind therapy when patients experienced a seizure cluster meeting study criteria. A second, open-label 5 mg dose of Nayzilam could be administered between 10 minutes and six hours after the first dose for persistent or recurrent seizure activity.

Nayzilam appeared to be safe and well-tolerated, and adverse events were generally consistent with the route of administration and pharmacology of the compound.

The trial met its primary objective, which assessed the proportion of patients whose seizures ended within 10 minutes of Nayzilam administration, and who did not have a recurrence of seizures between 10 minutes and six hours after administration.

“There is an unmet need for effective and convenient acute treatment of seizure clusters that can rapidly end ongoing seizures and potentially prevent or delay their reoccurrence,” Wren said.

Nayzilam was granted orphan drug and fast track designation by the FDA for the rescue treatment of seizures in patients who experience recurrent periods of high seizure activity.

The fast track designation allows for an accelerated process by the FDA to review treatments that could treat serious conditions and fill an unmet medical need.

Nayzilam was acquired by UCB from Proximagen in June. Since then, both companies have worked together to advance the NDA filing.