Abstract Accepted for Oral Presentation at the 31st Annual Meeting of the Associated Professional Sleep Societies LLC (APSS) in June 2017
DUBLIN, Ireland I April 24, 2017 I Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced positive top-line efficacy results from the global, double-blind, placebo-controlled, randomized-withdrawal, multicenter Phase 2/3 study evaluating Xyrem® (sodium oxybate) oral solution, CIII, in the treatment of cataplexy in pediatric patients with narcolepsy. Xyrem demonstrated statistically significant differences in the primary and key secondary efficacy endpoints that measured the change in the weekly number of cataplexy attacks, the Clinical Global Impression of Change scale (CGIc) for the severity of cataplexy, and the Epworth Sleepiness Scale (ESS) for children and adolescents (CHAD) score compared to placebo. The preliminary safety results were consistent with the results previously observed in Xyrem studies in adults and our Xyrem post-marketing experience.
“More than half of all narcolepsy patients report that their symptoms began as teenagers or in childhood. Although narcolepsy in children is characterized by the same symptoms as adults, pediatric narcolepsy is frequently under-recognized and under-diagnosed,” said Karen Smith, M.D., Ph.D., global head of research and development and chief medical officer of Jazz Pharmaceuticals. “We look forward to presenting the results from this large pivotal Xyrem pediatric study in narcolepsy at the APSS meeting in June. We expect to submit a supplemental NDA to the FDA in support of the use of Xyrem in pediatric patients in the fourth quarter of 2017, subject to completion of the full data analysis.”
About the Global Phase 2/3 EXPRESS1 Study
The EXPRESS study is a double-blind, placebo-controlled, randomized-withdrawal, multicenter study evaluating the efficacy and safety of Xyrem with an open-label pharmacokinetic evaluation and safety extension for at least one year in pediatric patients with narcolepsy with cataplexy. The study enrolled 106 patients aged seven to seventeen with narcolepsy with cataplexy. The study design included a titration period (if necessary), a Xyrem stable-dose period of two to three weeks, followed by a 1:1 randomization to either Xyrem or placebo for 2 weeks. After the completion of the double-blind, placebo-controlled treatment period, patients have the opportunity to receive Xyrem in an open-label treatment phase for at least one year. After a pre-planned interim analysis, efficacy on the primary endpoint was demonstrated. As a result, randomization to placebo was discontinued, and the study remains ongoing with open-label Xyrem treatment.
The primary efficacy endpoint was the change in the weekly number of cataplexy attacks from the last 2 weeks of the stable-dose period to the 2 weeks of the double-blind placebo-controlled treatment period. The key secondary endpoints measured the CGIc for cataplexy severity and the ESS (CHAD) score from the end of the stable-dose period to the end of the double-blind placebo-controlled treatment period.
About Narcolepsy
Narcolepsy is a debilitating neurological disorder characterized by excessive sleepiness, and the inability to regulate sleep-wake cycles normally. It affects an estimated one in 2,000 people in the United States, and more than half of narcolepsy patients report that their symptoms began as teenagers or in childhood.2 It is estimated that 50 percent or more patients with narcolepsy have not been diagnosed. Studies have shown it may take 10 years or more for people with narcolepsy to receive a correct diagnosis. Of the five key symptoms of narcolepsy, excessive daytime sleepiness and cataplexy are the most common symptoms.
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is an international biopharmaceutical company focused on improving patients’ lives by identifying, developing and commercializing meaningful products that address unmet medical needs. The company has a diverse portfolio of products and product candidates, with a focus in the areas of sleep and hematology/oncology. In these areas, Jazz Pharmaceuticals markets Xyrem® (sodium oxybate) oral solution, Erwinaze® (asparaginase Erwinia chrysanthemi) and Defitelio® (defibrotide sodium) in the U.S. and markets Erwinase® and Defitelio® (defibrotide) in countries outside the U.S. For more information, please visit www.jazzpharmaceuticals.com.
About Xyrem
Xyrem oral solution, CIII, is indicated for the treatment of cataplexy in narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in narcolepsy. Xyrem may only be dispensed to patients enrolled in the Xyrem REMS Program. Xyrem was first approved in the U.S. in 2002, based on clinical trial data in adults. The current USPI for Xyrem indicates that safety and effectiveness in pediatric patients have not been established. Therefore, subject to completion of the full data analysis, Jazz plans to submit a supplemental NDA to FDA in the fourth quarter of 2017, with revised proposed labeling to include these data, consistent with the Agency’s pediatric written request.
Reference:
1 EXPRESS stands for the Effect of Xyrem in Pediatric naRcolEpSy patientS
2 Dauvilliers Y, Montplaisir J, Molinari N, Carlander B, Ondze B, Besset A, Billiard M. Age at onset of narcolepsy in two large populations of patients in France and Quebec. Neurol 2001; 57:2029-2033.
SOURCE: Jazz Pharmaceuticals
Post Views: 28
Abstract Accepted for Oral Presentation at the 31st Annual Meeting of the Associated Professional Sleep Societies LLC (APSS) in June 2017
DUBLIN, Ireland I April 24, 2017 I Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced positive top-line efficacy results from the global, double-blind, placebo-controlled, randomized-withdrawal, multicenter Phase 2/3 study evaluating Xyrem® (sodium oxybate) oral solution, CIII, in the treatment of cataplexy in pediatric patients with narcolepsy. Xyrem demonstrated statistically significant differences in the primary and key secondary efficacy endpoints that measured the change in the weekly number of cataplexy attacks, the Clinical Global Impression of Change scale (CGIc) for the severity of cataplexy, and the Epworth Sleepiness Scale (ESS) for children and adolescents (CHAD) score compared to placebo. The preliminary safety results were consistent with the results previously observed in Xyrem studies in adults and our Xyrem post-marketing experience.
“More than half of all narcolepsy patients report that their symptoms began as teenagers or in childhood. Although narcolepsy in children is characterized by the same symptoms as adults, pediatric narcolepsy is frequently under-recognized and under-diagnosed,” said Karen Smith, M.D., Ph.D., global head of research and development and chief medical officer of Jazz Pharmaceuticals. “We look forward to presenting the results from this large pivotal Xyrem pediatric study in narcolepsy at the APSS meeting in June. We expect to submit a supplemental NDA to the FDA in support of the use of Xyrem in pediatric patients in the fourth quarter of 2017, subject to completion of the full data analysis.”
About the Global Phase 2/3 EXPRESS1 Study
The EXPRESS study is a double-blind, placebo-controlled, randomized-withdrawal, multicenter study evaluating the efficacy and safety of Xyrem with an open-label pharmacokinetic evaluation and safety extension for at least one year in pediatric patients with narcolepsy with cataplexy. The study enrolled 106 patients aged seven to seventeen with narcolepsy with cataplexy. The study design included a titration period (if necessary), a Xyrem stable-dose period of two to three weeks, followed by a 1:1 randomization to either Xyrem or placebo for 2 weeks. After the completion of the double-blind, placebo-controlled treatment period, patients have the opportunity to receive Xyrem in an open-label treatment phase for at least one year. After a pre-planned interim analysis, efficacy on the primary endpoint was demonstrated. As a result, randomization to placebo was discontinued, and the study remains ongoing with open-label Xyrem treatment.
The primary efficacy endpoint was the change in the weekly number of cataplexy attacks from the last 2 weeks of the stable-dose period to the 2 weeks of the double-blind placebo-controlled treatment period. The key secondary endpoints measured the CGIc for cataplexy severity and the ESS (CHAD) score from the end of the stable-dose period to the end of the double-blind placebo-controlled treatment period.
About Narcolepsy
Narcolepsy is a debilitating neurological disorder characterized by excessive sleepiness, and the inability to regulate sleep-wake cycles normally. It affects an estimated one in 2,000 people in the United States, and more than half of narcolepsy patients report that their symptoms began as teenagers or in childhood.2 It is estimated that 50 percent or more patients with narcolepsy have not been diagnosed. Studies have shown it may take 10 years or more for people with narcolepsy to receive a correct diagnosis. Of the five key symptoms of narcolepsy, excessive daytime sleepiness and cataplexy are the most common symptoms.
About Jazz Pharmaceuticals
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is an international biopharmaceutical company focused on improving patients’ lives by identifying, developing and commercializing meaningful products that address unmet medical needs. The company has a diverse portfolio of products and product candidates, with a focus in the areas of sleep and hematology/oncology. In these areas, Jazz Pharmaceuticals markets Xyrem® (sodium oxybate) oral solution, Erwinaze® (asparaginase Erwinia chrysanthemi) and Defitelio® (defibrotide sodium) in the U.S. and markets Erwinase® and Defitelio® (defibrotide) in countries outside the U.S. For more information, please visit www.jazzpharmaceuticals.com.
About Xyrem
Xyrem oral solution, CIII, is indicated for the treatment of cataplexy in narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in narcolepsy. Xyrem may only be dispensed to patients enrolled in the Xyrem REMS Program. Xyrem was first approved in the U.S. in 2002, based on clinical trial data in adults. The current USPI for Xyrem indicates that safety and effectiveness in pediatric patients have not been established. Therefore, subject to completion of the full data analysis, Jazz plans to submit a supplemental NDA to FDA in the fourth quarter of 2017, with revised proposed labeling to include these data, consistent with the Agency’s pediatric written request.
Reference:
1 EXPRESS stands for the Effect of Xyrem in Pediatric naRcolEpSy patientS
2 Dauvilliers Y, Montplaisir J, Molinari N, Carlander B, Ondze B, Besset A, Billiard M. Age at onset of narcolepsy in two large populations of patients in France and Quebec. Neurol 2001; 57:2029-2033.
SOURCE: Jazz Pharmaceuticals
Post Views: 28
