Approximately 1/3 of all epilepsy patients live with uncontrolled seizures. Of these, around 150,000 in the United States alone experience cluster seizures (otherwise known as acute-repetitive seizures, crescendo seizures, serial seizures, or seizure flurries). Cluster seizures are defined as acute episodes of seizures that occur consecutively within a short period of time. They are distinguishable from the patients typical pattern of seizures.
Cluster seizures are considered an emergency and rescue therapy is imperative. When untreated, the patient is at risk for neurological damage, physical injury, and status epilepticus. However, current treatment options are far from ideal because administration of the therapy during the crisis is so difficult. Unfortunately, this means that many patients do not receive the critical treatment they need.
However, it has just been announced that the FDA has approved a new therapy for cluster seizures which can be administered through the nose. It is the first new medication in the United States approved for cluster seizures in the last 20 years. The therapy is called Nayzilam.
Nayzilam is a nasal spray formulation of a benzodiazepine. It was approved as a single-use, short-term treatment for cluster seizures in epilepsy patients who are at least 12 years of age. It is the first and the only approved therapy that utilizes nasal delivery.
This nasal spray can be carried by the patient and administered by someone who is not a healthcare professional in times of crisis.
This newly approved therapy could significantly improve outcomes for epilepsy patients who experience cluster seizures and ultimately, lead to reduced complications down the line.
The Phase 3 clinical trial which led to this new approval was a randomized, placebo-controlled, double-blind investigation which was conducted in two different phases. The first phase was an open-label Test Dose Phase which evaluated the tolerability of the nasal spray in 292 epilepsy patients. Each participant received 2, 5mg doses of Nayzilam, 10 minutes apart.
The second phase of this trial was a Comparative Phase. There were 201 participants in total. 134 received Nayzilam (5mg) and 67 patients received placebo. Both groups of patients had the option to receive an unblinded dose of Nayzilam (5mg) if seizure activity persisted. This administration was given between 10 minutes and 6 hours after they received the first blinded dose.
The primary endpoint of the trial was termination of seizures within 10 minutes post administration and no seizure recurrence within 6 hours. For both of these evaluations, the Nayzilam group had statistically significant outcomes in comparison to the placebo group. Additionally, fewer patients treated with the drug experienced a seizure within 24 hours after they received the treatment.
The most common AEs associated with Nayzilam were headache, throat irritation, nasal discomfort, rhinorrhea, and somnolence. However, more serious side effects are possible so it’s important to read the full prescribing information available here.
This new approval is amazing news for those living with Dravet Syndrome, Lennox-Gastaut Syndrome, and other forms of epilepsy. It could improve not only short-term outcomes, but longterm outcomes for this entire patient community.
You can read more about this newly approved therapeutic option here.