This approval was based on the Phase III withdrawal study that showed clinically meaningful differences vs. placebo in change to the primary endpoint, Epworth Sleepiness Scale score.
The U.S. Food and Drug Administration (FDA) has approved Jazz Pharmaceuticals’ Xywav for idiopathic hypersomnia in adults. This unusual neurologic sleep disorder is marked by chronic excessive daytime inability to stay awake, leading to an “irrepressible need to sleep.” An estimated 37,000 people in the U.S. have idiopathic hypersomnia.
Xywav, an oral solution of calcium, magnesium, potassium, and sodium oxybate, is already approved for narcolepsy.
“We are excited that with today’s approval, Xywav will become the first and only medicine indicated to treat idiopathic hypersomnia, a unique medical condition that can have significant effects on the lives of those diagnosed with the condition. Xywav is a meaningful treatment for patients as demonstrated by the statistically significant results from the Phase III clinical trial,” said Bruce Cozadd, chairman and chief executive officer of Jazz.
“We are proud to build on our leadership in sleep medicine and, with this approval, are expanding beyond our Xywav narcolepsy indications to bring this treatment to adults living with idiopathic hypersomnia who currently have no FDA-approved options available. This milestone exemplifies our patient-focused R&D strategy and internal development capabilities and underscores oxybate as a key growth opportunity for Jazz. With this launch, we will have achieved our goal of five product launches in two years.”
This approval was based on the Phase III withdrawal study that demonstrated statistically significant and clinically meaningful differences versus placebo in change to the primary endpoint, Epworth Sleepiness Scale score. It also hit the mark for secondary endpoints of Patient Global Impression of Change and the Idiopathic Hypersomnia Severity Scale.
Over 5% of patients reported nausea, headache, dizziness, anxiety, insomnia, decreased appetite, hyperhidrosis, vomiting, diarrhea, dry mouth, parasomnia, somnolence, fatigue, and tremor. It can be given twice or once a night.
The drug comes with a Boxed Warning because it is a central nervous system (CNS) depressant that can be abused. As such, it is only prescribed by way of a restricted program called the Xywav and Xyrem REMS.
The drug received FDA Fast Track designation in September 2020 for idiopathic hypersomnia and also received Priority Review for its supplemental New Drug Application (sNDA) in April 2021. This is the second approval. The first was in July 2020 for cataplexy or excessive daytime sleepiness in patients with narcolepsy who are seven years or older.
“The clinical program for Xywav has demonstrated that this lower-sodium oxybate is an effective therapy for the treatment of idiopathic hypersomnia,” said Yves Dauvilliers, director of the Sleep Disorders Centre at the Gui de Chauliac Hospital in Montpellier, France, and the lead investigator of the Phase III trial.
“Having an FDA-approved treatment option that manages symptoms associated with idiopathic hypersomnia, including excessive daytime sleepiness and severe sleep inertia, is a significant step forward for patients. Xywav fulfills an unmet need for those diagnosed with this sleep disorder, offering them management of their debilitating symptoms.”
According to Balaji Prasad, an analyst with Barclays, Xywav is projected to bring in sales of $1.49 billion by the end of 2025, with $1.1 billion for narcolepsy and about $300 million for this new indication.
