FDA Accepts Filing for Investigational Narcolepsy Medication

March 26, 2020

The FDA has accepted for filing with priority review Jazz Pharmaceuticals’ new drug application for JZP-258. The investigational medicine targets cataplexy and excessive daytime sleepiness associated with narcolepsy.

The FDA has accepted for filing with priority review Jazz Pharmaceuticals’ new drug application for JZP-258. The investigational medicine targets cataplexy and excessive daytime sleepiness (EDS) associated with narcolepsy.

JZP-258 can be used in patients aged 7 or older who suffer from narcolepsy. The investigational medicine “is a novel oxybate product candidate with a unique composition of cations resulting in 92%, or approximately 1000 to 1500 mg, less sodium than Xyrem (sodium oxybate),” according to a press release announcing the FDA's decision. Calcium, magnesium, and potassium cations are included in JZP-258.

The goal date for an FDA decision on JZP-258 is July 21, 2020. Currently, Xyrem is the only product approved to treat EDS and cataplexy in the same age group.

Cataplexy, a specific symptom of narcolepsy, is classified as the sudden, brief loss of muscle tone with retained consciousness that can be triggered by strong emotions like surprise or anger. Episodes of cataplexy commonly involve certain muscle groups, such as the arms and legs, head and neck, or the face and jaw.

“We developed JZP-258 to be a safer and long-term treatment option for patients. JZP-258 represents between 1000 and 1500 mg daily reduction of sodium for patients currently treated with Xyrem, depending on the dose,” said Robert Iannone, MD, executive vice president of research and development at Jazz Pharmaceuticals.

There is consensus in the scientific community that reducing daily sodium intake is associated with reductions in blood pressure and cardiovascular disease risk, explains Iannone. Individuals with narcolepsy have an increased risk of comorbidities including obesity, hypertension, and diabetes.

“We believe that JZP-258 has the potential to be an important treatment option for patients living with the life-long condition of narcolepsy,” he said.

The medicine can also be used to treat idiopathic hypersomnia in adult patients.

Although the exact mechanism of action of JZP-258 is not fully understood, researchers hypothesize the therapeutic effects of JZP-258 on sleep/wake symptoms are mediated through modulation of GABAB receptors as patients sleep.