Drug Receives FDA Approval for Treating Narcolepsy in Pediatric Patients
Officials with the FDA have approved Jazz Pharmaceuticals' Xyrem (sodium oxybate) for the treatment of cataplexy and excessive daytime sleepiness (EDS) in pediatric patients aged 7 to 17 years with narcolepsy.
Officials with the FDA have approved Jazz Pharmaceuticals' Xyrem (sodium oxybate) for the treatment of cataplexy and excessive daytime sleepiness in pediatric patients aged 7 to 17 years with narcolepsy. The drug had previously been approved for use in adult patients.1
Xyrem is a central nervous system (CNS) depressant that was FDA-approved in 2002. The medication was indicated for treating cataplexy, a sudden and transient episode of muscle weakness accompanied by full conscious awareness, typically triggered by emotions such as laughing, crying, or terror, in adult patients with narcolepsy.1 Approximately 70% of people with narcolepsy have cataplexy.2
According to the FDA, Xyrem, either alone or in combination with other CNS depressants, may be associated with adverse reactions that include seizure, respiratory depression, decreases in the level of consciousness, coma, and death. Sodium oxybate is the sodium salt of gamma hydroxybutyrate (GHB).2 According to the FDA, GHB has the potential for abuse, such as in cases of sexual assault.1,2
The most common adverse reactions in pediatric patients were bed-wetting, nausea, headache, vomiting, weight decrease, decreased appetite and dizziness.1
In addition, adverse reactions have been identified during post-approval use of Xyrem. They include joint pain, decreased appetite, fall, fluid retention, hangover, headache, hypersensitivity, hypertension, memory impairment, excessive nighttime urination, panic attack, vision blurred and weight decreased. However, because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure, according to the FDA.1
Due to the risk of serious outcomes resulting from inappropriate prescribing, misuse, abuse and diversion, sodium oxybate is only available through a risk evaluation mitigation strategy programs.1
In granting its newest approval for Xyrem, the FDA granted the application Priority Review and Orhan Drug designations.1 A generic version of sodium oxybate was first approved by the agency in early 2017.2
- FDA approves new treatment of cataplexy and excessive daytime sleepiness in pediatric patients with narcolepsy- Drug Information Update [news release]. Silver Spring, MD; October 26, 2018: FDA Division of Drug Information. Accessed October 28, 2018.
- FDA approves a generic of Xyrem with a REMS Program [news release]. Silver Spring, MD; January 17, 2017: FDA website. https://www.fda.gov/Drugs/DrugSafety/ucm537281.htm. Accesed October 28, 2018.