Results from a phase 3 study of Xywav in adult patients with idiopathic hypersomnia exhibited its potential to address this unmet medical need.
Results from Jazz Pharmaceuticals’ phase 3 study of Xywav in adult patients with idiopathic hypersomnia exhibited its potential to address this unmet medical need. Future research is additionally being considered in related areas of neuroscience, such as essential tremor, said Robert Iannone, MD, MSCE, executive vice president of research and development for Jazz Pharmaceuticals.
AJMC®: Hello, I'm Matthew Gavidia. Today on the MJH Life Sciences’ Medical World News, The American Journal of Managed Care® is pleased to welcome Robert Iannone, executive vice president of research and development at Jazz Pharmaceuticals.
Jazz Pharmaceuticals announced positive top-line results from its phase 3 study of Xywav oral solution in adult patients with idiopathic hypersomnia. How was the study conducted, and what primary and secondary endpoints were assessed?
Iannone: Yeah, really exciting news for us recently to hear the positive results from the idiopathic hypersomnia trial. Just to remind you, this was a clinical trial–a Phase 3 trial, double-blind, placebo-controlled, and in what we call a randomized withdrawal design.
So, what we did was we took patients who had idiopathic hypersomnia, and we allowed all of those patients to go on to Xywav during about a 4-month period where they were permitted to titrate their dose up until they had a maximal effect. At the end of the what we call titration period and stable dosing period, patients were assessed for their daytime sleepiness using a scale called the Epworth Sleepiness Scale (ESS). So, we compare baseline to the end of that stable dosing period. And the results show that during that period, patients improved in their daytime sleepiness symptoms.
The next part of the design was to randomly withdraw half of the patients. As I said, it's blinded and placebo-controlled so in a blinded fashion, half of the patients were then converted over to placebo. Then, we again measured that excessive sleepiness during the day using the ESS.
We also measured a patient-reported outcome called the Patient Global Impression of Change, as well as another patient-reported outcome called the Idiopathic Hypersomnia Severity Score. What we observed is those patients who were randomly withdrawn to placebo, had worsening in their daytime sleepiness, but also in their 2 measures of patient-reported outcomes, while the patients who remained on Xywav continued to have the benefit.
Then at the end of that 2-week, randomized withdrawal period, patients were then allowed to return again to their Xywav treatment, and we saw again improvement in those symptoms. So, it was a really nice demonstration of improvement from severity, return closer to baseline for those getting placebo, and then again a return up to benefit from the treatment. And I would say overall, the results were really promising in terms of the magnitude of effect that we observed.
AJMC®: To build upon that, with positive results from the phase 3 trial and Fast Track Designation granted by the FDA on Xywav, what are the next steps for this indication? And what other sleep medications are in the pipeline for Jazz Pharmaceuticals?
Iannone: So, Matthew, as you mentioned, Xywav was granted Fast Track Designation for the indication of idiopathic hypersomnia. And that comes when there's a condition such as idiopathic hypersomnia that is severe in nature, and when a new therapy has the potential to address this unmet medical need. So, we were really pleased and feel it's appropriate that Xywav received this designation.
As you know, there are currently no approved therapies in the United States for idiopathic hypersomnia. And so these results with Xywav are really, really encouraging. So, with that, we're working closely with the FDA to find really the appropriate and fastest path to bring this new therapy to patients who desperately need it.
To the second part of your question, Jazz continues to be committed to research and development in the space of sleep medicine. While we don't have any other ongoing phase 3 trials with Xywav, in particular, we're continuing to consider additional research in the area of sleep and narcolepsy, as well as expanding into other related areas of neuroscience, such as essential tremor.
AJMC®: Lastly, do you have any other concluding thoughts?
Iannone: Just that Jazz is really proud to be partnering with the American Heart Association to be providing important educational content for patients who are struggling with sleep or have sleep disorders, and that we after 15 years of working in the sleep field continue to be committed to research and development for patients who have sleep disorders.
AJMC®: To learn more, visit our website at AJMC.com. I’m Matthew Gavidia, thanks for joining us!