FDA Approves Sodium Oxybate for Cataplexy & Excessive Daytime Sleepiness in Pediatric Narcolepsy

Sodium oxybate (Xyrem) has been approved by the US Food and Drug Administration (FDA) for the treatment of cataplexy and excessive daytime sleepiness in pediatric patients aged 7 to 17 years old with narcolepsy.

Sodium oxybate is a central nervous system depressant administered as an oral solution and can be given either alone or in combination with other central nervous system depressants. These can include opioid analgesics, benzodiazepines, sedating antidepressants or antipsychotics, sedating anti-epileptic drugs, general anesthetics, muscle relaxants, and/or illicit central nervous system depressants.

The recommended dose for sodium oxybate is 0.5 g/mL, which may only be dispensed to patients enrolled in the XYREM REMS Program because of the risk of serious outcomes that can occur from inappropriate prescribing, misuse, abuse, and diversion.

Adverse reactions, such as seizure, respiratory depression, decreases in the level of consciousness, coma, and death, may be associated with the use of sodium oxybate. Additional risks that have been recorded by both voluntary and involuntary users (eg, assault victims) include the rapid onset of sedation coupled with amnesia, particularly when combined with alcohol. When using sodium oxybate, patients should not drink alcohol.

In patients being treated with sedative-hypnotic agents and those with succinic semialdehyde dehydrogenase deficiency, sodium oxybate is contraindicated.

Enuresis (bed-wetting), nausea, headache, vomiting, weight decrease, decreased appetite, and dizziness are the most common adverse reactions in pediatric patients.

During post-approval use of sodium oxybate, arthralgia (joint pain), decreased appetite, fall, fluid retention, hangover, headache, hypersensitivity, hypertension, memory impairment, nocturia (excessive nighttime urination), panic attack, vision blurred, and weight decrease were recorded as adverse reactions. However, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure since these reactions are reported voluntarily from a population of uncertain size.

Previously, the FDA granted a priority review and orphan drug designation to sodium oxybate for the recently approved indication. In addition, the central nervous system depressant was approved in 2002 for the treatment of cataplexy in adult patients with narcolepsy.