Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication Xyrem is indicated for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy. Xyrem is a central nervous system depressant indicated for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years of age and older with narcolepsy ( 1 ). FDA label
Also known as Xyrem (sodium oxybate) oral solution, Sodium Oxybate
Xyrem · 7 trials · 3 indications
Double-blind comparison of the change in weekly number of cataplexy attacks from the last 2 weeks of the Stable Dose Period to the 2 weeks of the Double-blind Treatment Period.
Number of subjects reporting adverse events.
Percentage of pain VAS responders. Subjects with a \>= 30% reduction in pain VAS from baseline to endpoint (week 14) were considered responders; all other subjects were considered non-responders. Missing data were handled using BOCF (Baseline Observation Carried Forward). The pain VAS ranges from 0 (no pain) to 100 (worst imaginable pain). The pain VAS was collected morning, afternoon and evening. Baseline is the average value recorded for the measure during the week prior to the end-of-baseline visit. Endpoint is the average value recorded for the measure during the week prior to week 14.
Number of Subjects with treatment-emergent adverse events.
Average Respiratory Rate at 1 year.
The Maintenance of Wakefulness Test consisted of four 20 minute tests of the patient's ability to remain awake in soporific conditions. The Mean change from baseline to week 8 in the average MWT number of minutes until sleep onset was the primary endpoint.
The percentage of participants who met all 3 of the following criteria: Reduction of \>=20% from baseline to week 8 in both PVAS \& FIQ total score and PGI-C response of "very much better" or "much better". Analysis was based on LOCF (Last Observation Carried Forward) data. The PVAS ranges from 0 (no pain) to 100 (worst imaginable pain). The FIQ ranges from 0 (best function) to 100 (worst function). PGI-C is a 7 point likert scale measuring change in the participant's fibromyalgia symptoms that ranges from "very much worse" to "very much better"
| Arm | Type | Description |
|---|---|---|
| Xyrem | EXPERIMENTAL | Active Xyrem at a dose ≤9 g/night |
| Xyrem Placebo | PLACEBO_COMPARATOR | Xyrem placebo at a volume and regimen equivalent to the stable dose of Xyrem. |
| 1 | EXPERIMENTAL | - |
| 2 | EXPERIMENTAL | - |
| Group 1. | EXPERIMENTAL | Xyrem + Modafinil Placebo |
| Group 2: | PLACEBO_COMPARATOR | Xyrem Placebo + Modafinil Placebo |
| Group 3 | ACTIVE_COMPARATOR | Xyrem Placebo + Modafinil at established dose |
| Group 4: | EXPERIMENTAL | Xyrem + Modafinil at established dose |
| 3 | PLACEBO_COMPARATOR | Placebo (one of two doses matching active treatment by volume). |
| Name | Type | Description |
|---|---|---|
| Xyrem | DRUG | - |
| Xyrem® | DRUG | flexible dosing |
| placebo | DRUG | Oral Solution |
| Xyrem (sodium oxybate) oral solution | DRUG | Xyrem (sodium oxybate) oral solution |
| Xyrem Placebo | DRUG | Xyrem Placebo oral solution 12 ml per day for 4 weeks and 18 ml per day for another 4 weeks. |
| Modafinil at established dose | DRUG | Modafinil oral capsules at 200 to 600 mg per day for 8 weeks. |
| Modafinil (Placebo) | DRUG | Modafinil Placebo oral capsules 1 to 3 capsules per day for 8 weeks. |
Inclusion Criteria: 1. Male or female subjects aged 7-16 years at Visit 2 for subjects on Xyrem at study entry and at Visit 1.1 for Xyrem-naïve subjects (to ensure subjects are \<18 years of age at the end of the study) 2. Have a primary diagnosis of narcolepsy with cataplexy that meets Internation...
Top 1 of 2 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Axsome Therapeutics, Inc. | AXSM | 2 | PHASE3 | AXS-14 |
Xyrem is studied for narcolepsy, narcolepsy with cataplexy, and fibromyalgia. Its clinical program includes a pediatric Phase 3 trial in narcolepsy with cataplexy and three Phase 3 trials in adults with fibromyalgia. Xyrem is a small molecule developed by Jazz Pharmaceuticals plc.
Xyrem is developed by Jazz Pharmaceuticals plc, which trades under the ticker JAZZ. The company sponsors the clinical program for the drug across narcolepsy with cataplexy and fibromyalgia.
Xyrem has been evaluated in Phase 3 clinical development. Four Phase 3 trials are recorded, all completed, with no active trials ongoing. The studies enrolled a total of 1,876 participants and were randomized, double-blind, and placebo-controlled.
Xyrem has been studied in four completed Phase 3 trials: NCT02221869 in pediatric narcolepsy with cataplexy, and NCT00423813, NCT00423605, and NCT00371137 in adults with fibromyalgia. The fibromyalgia trials enrolled 573, 560, and 548 participants respectively, and the narcolepsy trial enrolled 106.
Yes. Xyrem is the brand name for sodium oxybate oral solution, also written as Xyrem (sodium oxybate) oral solution. Searches for either name refer to the same Jazz Pharmaceuticals product and its clinical program in narcolepsy with cataplexy and fibromyalgia.
The Xyrem Phase 3 trials ran across North America and Europe. The pediatric narcolepsy with cataplexy study took place in the United States, France, Italy, and Netherlands, while the fibromyalgia trials enrolled participants in the United States and several European countries including Germany, Spain, and the United Kingdom.