Jazz Pharmaceuticals Announces FDA Approval of Xyrem® (sodium oxybate) for the Treatment of Cataplexy or Excessive Daytime Sleepiness in Pediatric Narcolepsy Patients
"Narcolepsy is often misunderstood, misrepresented, misdiagnosed and underdiagnosed, especially in children," said
"Xyrem is the only
The efficacy of Xyrem for the treatment of cataplexy or
Participants eligible for the study could either have been taking Xyrem or be Xyrem-naïve at study entry. Xyrem-naïve participants underwent open-label titration to reach a tolerable and effective dose. Following a stable dose period, all participants underwent a two week, double-blind, randomized-withdrawal period and were randomly assigned to either remain on Xyrem at their stable dose, or to receive placebo. The primary efficacy endpoint was the change in weekly number of cataplexy attacks, from the stable-dose period (baseline) to the end of the double-blind period. The change in
Participants who were randomized to placebo (withdrawn from Xyrem (32 [51%] of 63 patients)) had increased weekly cataplexy attacks (median increase of 12·7 attacks per week [Q1, Q3=3·4, 19·8]) when compared with those who continued treatment with Xyrem (median increase of 0·3 attacks per week [Q1, Q3 = –1·0, 2·5]; p<0·0001).
The safety profile of Xyrem in children and adolescents in this study was similar to that reported in adults, and no new safety concerns were identified following the use of Xyrem for up to one year. The open-label portion of the study is still ongoing. As of February 2017 (the time of the data cut), the most common Treatment-Emergent Adverse Events (TEAEs) (>5%) were enuresis, nausea, vomiting, headache, decreased weight, decreased appetite, nasopharyngitis and dizziness. Two serious TEAEs occurred (acute psychosis which was severe and suicidal ideation which was moderate in severity).
For the data cut, safety assessments included measures of anxiety (Multidimensional Anxiety Scale for Children 10-item [MASC-10]), depressive symptoms (Children's Depression Inventory 2nd Edition Self-Report Short Version [CDI 2:SR(S)]) and suicidality (Columbia-Suicide Severity Rating Scale [C-SSRS]), in addition to TEAEs. T-scores on the MASC-10 were within the average range throughout the study in participants who were Xyrem-naïve and on-Xyrem at study entry. T-scores on the CDI 2:SR(S) were within the average range throughout the study in participants who were Xyrem-naïve and on-Xyrem at study entry; a slight downward trend was observed in mean CDI 2:SR(S) T-scores over time.
Results from the Phase 2/3 EXPRESS study were published in The
The Micromedex DRUGDEX® monograph for Xyrem contains a Class IIA recommendation (recommended, in most cases) for use to treat cataplexy in narcolepsy in the pediatric patient population. The Micromedex DRUGDEX® is one of several statutorily named compendia in
Xyrem oral solution, CIII, is the only product approved by the
IMPORTANT SAFETY INFORMATION
WARNING: Taking XYREM with other CNS depressants such as medicines used to make you or your child fall asleep, including opioid analgesics, benzodiazepines, sedating antidepressants, antipsychotics, sedating anti-epileptic medicines, general anesthetics, muscle relaxants, alcohol, or street drugs, may cause serious medical problems, including trouble breathing (respiratory depression), low blood pressure (hypotension), changes in alertness (drowsiness), dizziness (syncope), and death.
XYREM is a form of gamma hydroxybutyrate (GHB). Abuse or misuse of illegal GHB alone or with other drugs that cause changes in alertness (or consciousness) has caused serious side effects. These effects include seizures, trouble breathing (respiratory depression), changes in alertness (drowsiness), coma, and death.
Because of these risks, you have to go through the XYREM REMS Program to have your or your child's prescription for XYREM filled.
Do not take XYREM if you take or your child takes other sleep medicines or sedatives (medicines that cause sleepiness), drink alcohol, or have a rare problem called succinic semialdehyde dehydrogenase deficiency.
Keep XYREM in a safe place to prevent abuse and misuse. Selling or giving away XYREM may harm others, and is against the law. Tell your doctor if you have ever abused or been dependent on alcohol, prescription medicines, or street drugs.
Anyone who takes XYREM should not do anything that requires them to be fully awake or is dangerous, including driving a car, using heavy machinery, or flying an airplane, for at least 6 hours after taking XYREM. Those activities should not be done until you know how XYREM affects you or your child.
XYREM can cause serious side effects, including the following:
- Breathing problems, including slower breathing, trouble breathing, and/or short periods of not breathing while sleeping (sleep apnea). People who already have breathing or lung problems have a higher chance of having breathing problems when they use XYREM.
- Mental health problems, including confusion, seeing or hearing things that are not real (hallucinations), unusual or disturbing thoughts (abnormal thinking), feeling anxious or upset, depression, or thoughts of killing yourself or trying to kill yourself. Tell your doctor if you or your child have or had depression or have tried to harm yourself. Call your doctor right away if you have or your child has symptoms of mental health problems.
- Sleepwalking. Sleepwalking can cause injuries. Call your doctor if you or your child starts sleepwalking. Your doctor should check you or your child.
Tell your doctor if you are or your child is on a salt-restricted diet or if you have or your child has high blood pressure, heart failure, or kidney problems. XYREM contains a lot of sodium (salt) and may not be right for you or your child.
The most common side effects of XYREM include nausea, sleepiness, dizziness, vomiting, bedwetting, and tremor (in adults). In pediatric patients, headache, decreased appetite, and weight decrease were also common. Your side effects may increase when you take higher doses of XYREM. XYREM can cause physical dependence and craving for the medicine when it is not taken as directed. These are not all the possible side effects of XYREM.
You are encouraged to report negative side effects of prescription drugs to the
Please find full prescribing information here: http://pp.jazzpharma.com/pi/xyrem.en.USPI.pdf
Narcolepsy is a chronic neurologic condition (involving nerve cells and chemicals in the brain) in which the brain is not able to control sleep-wake cycles normally.1 Patients with narcolepsy have excessive daytime sleepiness (
1. Narcolepsy Fact Sheet.
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4. Pelayo R, Lopes MC. Narcolepsy. In: Lee-Chiong TL. Sleep: A Comprehensive Handbook. Hoboken:
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6. Morrish E, King MA, Smith IE, Shneerson JM. Factors associated with a delay in the diagnosis of narcolepsy. Sleep Med. 2004;5(1):37-41.
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