Study results for XYWAV in adults with narcolepsy

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The phase 3 clinical study

  • The efficacy and safety of XYWAV were studied in 201 adults aged 18-70 years with narcolepsy who had at least 14 cataplexy attacks in a typical 2-week period before they took any treatment*
  • All people in the clinical study started treatment on XYWAV. Some participants continued taking their stimulant or wake-promoting agent along with XYWAV
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Study design of the XYWAV clinical trial for narcolepsy over a 16-week time period
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Study design of the XYWAV clinical trial for narcolepsy over a 16-week time period

*Not everyone completed each part of the study. Of the 201 patients, 134 were included in the 2-week period that measured how well XYWAV worked.

Results from the XYWAV clinical trial

During the 2-week period of the study that measured how well XYWAV worked:

Patients on XYWAV showed less of an increase in cataplexy attacks

In people who stopped taking XYWAV,

cataplexy increased by an average of:
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11.5 attacks per week

In people who continued taking XYWAV,

cataplexy increased by an average of:
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0.1 attacks per week
Patients on XYWAV showed less of an increase in excessive daytime sleepiness (EDS)

In people who stopped taking XYWAV,

daytime sleepiness increased by an average of:
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3.0 points

In people who continued taking XYWAV,

daytime sleepiness increased by an average of:
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0.0 points

The Epworth Sleepiness Scale (ESS) is intended to measure EDS, a symptom of narcolepsy. It is not intended to make a narcolepsy diagnosis.

See where your daytime sleepiness falls on the Epworth Sleepiness Scale (ESS) and share your results with your doctor

Download ESS

After the phase 3 study was completed, a separate analysis of the data examined cataplexy-free days* during the study

This information is from a separate study, also called a post-hoc analysis, that took place after the original study that looked at patients’ daily cataplexy frequency diaries to learn how often cataplexy occurred.

Cataplexy-free days are days in which a patient did not experience a cataplexy attack.

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Post hoc analysis of cataplexy-free data in week 1 and week 2 of the clinical trial for XYWAV and narcolepsy
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Cataplexy-free data in week 1 and week 2 of the clinical trial for XYWAV and narcolepsy
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Cataplexy-free data in week 1 and week 2 of the clinical trial for XYWAV and narcolepsy

This post-hoc analysis was exploratory, which means it cannot be used to show how well XYWAV works. An additional analysis may be warranted.
*Based on the median, which is the middle number in a data set.

The phase 4 clinical study

The DUET study evaluated the effect of XYWAV on nighttime sleep and daytime symptoms in patients with narcolepsy

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Study design for XYWAV clinical study in narcolepsy, analyzing effect on daytime and nighttime symptoms
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Study design for XYWAV clinical study in narcolepsy, analyzing effect on daytime and nighttime symptoms

This information is from a phase 4 study, which takes place after a treatment has been FDA-approved.

All 55 participants in the study knew they were taking XYWAV (this is called an "open-label" study). There was also no placebo group for comparison. As a result, it cannot be determined whether changes were related to treatment or to other factors. This study is descriptive and should not be used to show how well XYWAV works.

*Period of time where participants stopped taking any oxybates to help establish a baseline.

Primary endpoint: change in excessive daytime sleepiness (EDS)

This information is from a phase 4 study, which takes place after a treatment has been FDA-approved.

34 participants completed the ESS at baseline and at the end of treatment. All participants knew they were taking XYWAV (this is called an "open-label" study). There was also no placebo group for comparison. As a result, it cannot be determined whether changes were related to treatment or to other factors. This study is descriptive and should not be used to show how well XYWAV works.

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In an open-label, single-arm trial, the average ESS score at the start of the study was 16. At the end of treatment, the average score was 9.

The Epworth Sleepiness Scale (ESS) measures a person's general level of daytime sleepiness. This study looked at the ESS scores when people started the study (16 points on average) and the scores once they had been on XYWAV (9 points on average) and found a change of –7.7.
95% confidence interval (CI) of -9.6 to -5.8. This means there is 95% confidence that the true population mean change lies between these numbers.
There was a standard error of 0.9. Standard error measures how much a result would likely change if you repeated the study with a different set of people.

Sleep stages are the different phases your brain and body cycle through when you sleep. Each stage—ranging from light sleep to deep sleep and REM sleep—plays a role in helping you rest and recharge.

DUET secondary endpoint: transitions between sleep to light sleep or wake
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In an open-label, single-arm trial, the average change in number of transitions from sleep to light sleep or wake from baseline to end of study was -13.1
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In an open-label, single-arm trial, the average change in number of transitions from sleep to light sleep or wake from baseline to end of study was -13.1
  • This information is from a phase 4 study, which takes place after a treatment has been FDA-approved
  • 34 participants had sleep stage transitions measured at baseline and end of treatment. All participants knew they were taking XYWAV (this is called an "open-label" study). There was also no placebo group for comparison. As a result, it cannot be determined whether changes were related to treatment or to other factors. This study is descriptive and show not be used to show how well XYWAV works

This study looked at the number of transitions between sleep to light sleep or wake when people started the study (54.6 total transitions on average) and the number of transitions once they had been on XYWAV (41.6 total transitions on average) and found a change of -13.1.
95% confidence interval (CI) of –19.0 to –7.1. This means there is 95% confidence that the true population mean change lies between these numbers.
There was a standard error of 2.9. Standard error measures how much a result would likely change if you repeated the study with a different set of people.
REM=rapid eye movement.

DUET secondary endpoint: change in number of nighttime awakenings
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In an open-label, single-arm trial, the average change in number of awakenings from baseline to end of study was 3.2
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In an open-label, single-arm trial, the average change in number of awakenings from baseline to end of study was 3.2
  • This information is from a phase 4 study, which takes place after a treatment has been FDA-approved
  • 34 participants had the number of nighttime awakenings measured at baseline and the end of treatment. All participants knew they were taking XYWAV (this is called an "open-label" study). There was also no placebo group for comparison. As a result, it cannot be determined whether changes were related to treatment or to other factors. This study is descriptive and show not be used to show how well XYWAV works

This study looked at the number of nighttime awakenings when people started the study (13.9 awakenings on average) and the number of awakenings once they had been on XYWAV (10.8 awakenings on average) and found a change of -3.2.
95% confidence interval (CI) of –5.0 to –1.3. This means there is 95% confidence that the true population mean change lies between these numbers.
There was a standard error of 0.9. Standard error measures how much a result would likely change if you repeated the study with a different set of people.

See where your daytime sleepiness falls on the Epworth Sleepiness Scale (ESS) and share your results with your doctor

Download ESS

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