Jazz Pharmaceuticals Announces Positive Top-line Results from Phase 3 Study of Xywav™ (calcium, magnesium, potassium, and sodium oxybates) Oral Solution in Adult Patients with Idiopathic Hypersomnia
DUBLIN, Oct. 8, 2020 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced positive top-line results from the Phase 3 double-blind, multicenter, placebo-controlled, randomized withdrawal study evaluating the efficacy and safety of an investigational use of Xywav™ (calcium, magnesium, potassium, and sodium oxybates) oral solution in adult patients with idiopathic hypersomnia.
Patients entering the study had excessive daytime sleepiness typical of the idiopathic hypersomnia population. All patients were treated with Xywav during the open-label titration period and clinically meaningful improvements in the Epworth Sleepiness Scale (ESS) were observed.
The primary endpoint of ESS and the key secondary endpoints of Patient Global Impression of Change (PGIc) and Idiopathic Hypersomnia Severity Scale (IHSS) were measured during the randomized withdrawal portion of the trial, which included 115 patients. Those who were administered Xywav showed clinically meaningful maintenance of efficacy for ESS, PGIc and IHSS, and there were highly statistically significant worsenings in patients administered placebo compared with Xywav for ESS (p-value <0.0001), PGIc (p-value <0.0001) and IHSS (p-value <0.0001).
The safety profile in this study was consistent with the known safety profile of Xywav with no new safety signals observed in this population.
"We are excited by these compelling results and the magnitude of improvement observed in the study, in particular for people living with idiopathic hypersomnia who currently have no approved treatment option. We are deeply grateful to the patients and investigators who participated in the study, and look forward to working quickly with the FDA to make Xywav available to patients as soon as possible," said Robert Iannone, M.D., M.S.C.E., executive vice president, research and development of Jazz Pharmaceuticals. "For more than 15 years, Jazz has been at the forefront of sleep medicine. Our purpose is to innovate to transform the lives of patients and we are committed to bringing new options for people living with serious sleep disorders where there are no or limited treatments available."
Jazz will submit the Phase 3 study data for presentation at an upcoming medical meeting and these data will be included in the planned submission of a supplemental New Drug Application (sNDA) to the FDA as early as the first quarter of 2021. The FDA granted Fast Track designation to Xywav in September 2020.
Idiopathic hypersomnia is a chronic, neurological disorder that is characterized by excessive sleepiness, an uncontrollable need to sleep or daytime sleepiness that persists for at least 3 months even with adequate or prolonged nighttime sleep.
"There is significant need for greater awareness of idiopathic hypersomnia, which can severely impact a person's daily life, and can often be mis-diagnosed or undiagnosed over a substantial period of time," said Yves Dauvilliers, MD, Director, Sleep Disorders Centre, Gui de Chauliac Hospital in Montpellier, France and lead investigator of the Phase 3 study. "Currently, there are no approved treatment options for idiopathic hypersomnia, and these data are a welcome advance for patients.3"
Xywav was approved in July 2020 by the FDA for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy.1,2 Xywav is not currently approved by regulatory authorities for the treatment of idiopathic hypersomnia.
About the Phase 3 Study in Idiopathic Hypersomnia
The study design included a titration and optimization period of up to 14 weeks, a Xywav stable-dose period of two weeks, followed by a 1:1 randomization to either Xywav or placebo for 2 weeks. After the completion of the double-blind, placebo-controlled treatment period, patients entered a 24-week open-label safety extension period. More information about the study design is available at www.clinicaltrials.gov (identifier: NCT03533114).
About Idiopathic Hypersomnia
More information about Xywav, including Full Prescribing Information and Medication Guide, is available here. <http://pp.jazzpharma.com/pi/xywav.en.USPI.pdf>
About Xywav™ (calcium, magnesium, potassium, and sodium oxybates) oral solution
Important Safety Information
WARNING: Taking XYWAV with other central nervous system (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), fainting (syncope), and death.
The active ingredient of XYWAV 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. Call your doctor right away if you or your child has any of these serious side effects.
Because of these risks, you have to go through the XYWAV and XYREM REMS Program to have your or your child's prescription for XYWAV filled.
Do not take XYWAV if you take or your child takes other sleep medicines or sedatives (medicines that cause sleepiness), drinks alcohol, or has a rare problem called succinic semialdehyde dehydrogenase deficiency.
Keep XYWAV in a safe place to prevent abuse and misuse. Selling or giving away XYWAV 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 XYWAV 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 XYWAV. Those activities should not be done until you know how XYWAV affects you or your child.
XYWAV can cause serious side effects, including the following:
The most common side effects of XYWAV in adults include headache, nausea, dizziness, decreased appetite, parasomnia (a sleep disorder that can include abnormal dreams, abnormal rapid eye movement (REM) sleep, sleep paralysis, sleep talking, sleep terror, sleep-related eating disorder, sleep walking, and other abnormal sleep-related events), diarrhea, excessive sweating (hyperhidrosis), anxiety and vomiting.
The most common side effects of XYWAV in children include bedwetting, nausea, headache, vomiting, weight decrease, decreased appetite, and dizziness.
XYWAV 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 XYWAV.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
About Jazz Pharmaceuticals plc
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SOURCE Jazz Pharmaceuticals plc
Company Codes: NASDAQ-NMS:JAZZ