DUBLIN, Oct. 31, 2024 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced that yesterday, the U.S. District Court for the District of Columbia ("Court") ruled in favor of the Food and Drug Administration ("FDA") in a suit brought by Jazz Pharmaceuticals Inc. ("Jazz") under the Administrative Procedure Act regarding the FDA's approval of LUMRYZTM, the first and only once-at-bedtime oxybate treatment for cataplexy or excessive daytime sleepiness in adults with narcolepsy. With this ruling, the approval of LUMRYZ is upheld based on the FDA's determination that LUMRYZ is clinically superior to Jazz's twice-nightly oxybate products.
Following the FDA's final approval and grant of Orphan Drug Exclusivity ("ODE") to LUMRYZ in May 2023, Jazz filed a complaint against the FDA claiming that LUMRYZ's approval was not in alignment with the Orphan Drug Act. Avadel CNS Pharmaceuticals, LLC ("Avadel CNS") intervened to defend the FDA's actions and successfully argued to uphold approval of LUMRYZ.
"We are pleased with the Court's ruling in favor of the FDA's clinical superiority determination for LUMRYZ in conjunction with final approval for use in adults with narcolepsy. With the Court's decision, LUMRYZ will continue to be available to the narcolepsy community and retain its Orphan Drug Exclusivity," said Greg Divis, Chief Executive Officer at Avadel Pharmaceuticals. "Yesterday's ruling further solidifies LUMRYZ's unique once-at-bedtime dosing schedule as a major contribution to patient care and enables us to continue executing on our commercial launch and expanding LUMRYZ's reach within the narcolepsy community."
About LUMRYZ (sodium oxybate) for extended-release oral suspension
LUMRYZ is an extended-release sodium oxybate medication approved by the FDA on May 1, 2023, as the first and only once-at-bedtime treatment for cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. On October 16, 2024, LUMRYZ was additionally approved as a once-at-bedtime treatment for cataplexy or EDS in pediatric patients 7 years of age and older with narcolepsy.
The FDA approval of LUMRYZ was supported by results from REST-ON, a randomized, double-blind, placebo-controlled, pivotal Phase 3 trial in adults with narcolepsy. LUMRYZ demonstrated statistically significant and clinically meaningful improvements in the three co-primary endpoints: EDS (MWT), clinicians' overall assessment of patients' functioning (CGI-I), and cataplexy attacks, for all three evaluated doses when compared to placebo.
With its original approval in May 2023, the FDA granted 7 years of Orphan Drug Exclusivity to LUMRYZ for the treatment of cataplexy or EDS in adults with narcolepsy due to a finding of clinical superiority of LUMRYZ relative to currently available oxybate treatments. In particular, the FDA found that LUMRYZ makes a major contribution to patient care over currently available, twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose. Similarly, with its pediatric approval in October of 2024, the FDA granted another 7 years of Orphan Drug Exclusivity to LUMRYZ for the treatment of cataplexy or EDS in patients 7 years and older with narcolepsy due to a finding of clinical superiority of LUMRYZ relative to currently available oxybate treatments. In particular, the FDA found there that LUMRYZ's dosing makes a major contribution to patient care by providing an opportunity to minimize sleep fragmentation and disruption of sleep architecture in a way that is not possible for a patient on a twice-nightly dosing regimen of oxybate.
About Avadel Pharmaceuticals plc
Avadel Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical company focused on transforming medicines to transform lives. Our approach includes applying innovative solutions to the development of medications that address the challenges patients face with current treatment options. Avadel's commercial product, LUMRYZ, was approved by the U.S. Food & Drug Administration (FDA) as the first and only once-at-bedtime oxybate for the treatment of cataplexy or excessive daytime sleepiness (EDS) in patients 7 years and older with narcolepsy. For more information, please visit .
IMPORTANT SAFETY INFORMATION
WARNING: Taking LUMRYZ (sodium oxybate) with other central nervous system (CNS) depressants, such as medicines used to make you 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 LUMRYZ (sodium oxybate) is a form of gamma hydroxybutyrate (GHB), a controlled substance. Abuse or misuse of illegal GHB alone or with other CNS depressants (drugs that cause changes in alertness or consciousness) have 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 have any of these serious side effects.
Because of these risks, LUMRYZ is available only by prescription and filled through certified pharmacies in the LUMRYZ REMS. You must be enrolled in the LUMRYZ REMS to receive LUMRYZ. Further information is available at or by calling 1-877-453-1029. |
INDICATIONS
LUMRYZ (sodium oxybate) for extended-release oral suspension is a prescription medicine used to treat the following symptoms in patients 7 years of age and older with narcolepsy:
Do not take LUMRYZ if you take or your child takes other sleep medicines or sedatives (medicines that cause sleepiness), drink alcohol or have a rare problem called succinic semialdehyde dehydrogenase deficiency.
Keep LUMRYZ in a safe place to prevent abuse and misuse. Selling or giving away LUMRYZ may harm others and is against the law. Tell your doctor if you or your child have ever abused or been dependent on alcohol, prescription medicines or street drugs.
Anyone who takes LUMRYZ 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 six (6) hours after taking LUMRYZ. Those activities should not be done until you know how LUMRYZ affects you.
Falling asleep quickly, including while standing or while getting up from the bed, has led to falls with injuries that have required some people to be hospitalized.
LUMRYZ can cause serious side effects, including the following:
Breathing problems, including slower breathing, trouble breathing and/or short periods of not breathing while sleeping (e.g., sleep apnea). People who already have breathing or lung problems have a higher chance of having breathing problems when they take LUMRYZ.
Mental health problems, including confusion, seeing or hearing things that are not real (hallucinations), unusual or disturbing thoughts (abnormal thinking), feeling anxious or upset, depression, thoughts of killing yourself or trying to kill yourself, increased tiredness, feelings of guilt or worthlessness and difficulty concentrating. Tell your doctor if you or your child have or had depression or have tried to harm yourself. Call your doctor right away if you or your child have symptoms of mental health problems or a change in weight or appetite.
Sleepwalking. Sleepwalking can cause injuries. Call your doctor if you or your child start sleepwalking.
Tell your doctor if you or your child are on a salt-restricted diet or have high blood pressure, heart failure or kidney problems. LUMRYZ contains a lot of sodium (salt) and may not be right for you.
The most common side effects of LUMRYZ in adults include nausea, dizziness, bedwetting, headache and vomiting. Your side effects may increase when you take higher doses of LUMRYZ. The most common side effects in children include nausea, bedwetting, vomiting, headache, decreased weight, decreased appetite, dizziness, and sleepwalking. LUMRYZ 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 LUMRYZ.
For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit , or call 1-800-FDA-1088.
Please see full Prescribing Information, including BOXED Warning.
都柏林，2024年10月31日（GLOBE NEWSWIRE）——专注于改造药物以改变生活的生物制药公司Avadel Pharmicals plc（纳斯达克股票代码：AVDL）昨天宣布，美国哥伦比亚特区地方法院（“法院”）在爵士制药公司（“Jazz”）根据《行政诉讼法》提起的诉讼中作出了有利于美国食品药品监督管理局（“FDA”）的裁决美国食品和药物管理局批准了LumryzTM，这是第一种也是唯一一种针对成人猝倒或日间过度嗜睡的睡前氧气疗法发作性睡病。该裁决维持了对LUMRYZ的批准，因为美国食品药品管理局认定LUMRYZ在临床上优于Jazz每晚两次的含氧产品。
继美国食品药品管理局于2023年5月最终批准并授予LUMRYZ孤儿药独家经营权（“ODE”）之后，Jazz对美国食品药品管理局提起诉讼，声称LUMRYZ的批准不符合《孤儿药法》。Avadel CNS Pharmicals, LLC（“Avadel CNS”）进行了干预，为美国食品药品管理局的行为辩护，并成功地主张维持对LUMRYZ的批准。
“我们对法院的裁决感到满意，该裁决支持美国食品药品管理局对LUMRYZ的临床优势裁决，同时最终批准用于发作性睡病成人。根据法院的裁决，LUMRYZ将继续向发作性睡病界开放，并保留其孤儿药独家经营权。” Avadel Pharmicals首席执行官格雷格·迪维斯说。“昨天的裁决进一步巩固了LUMRYZ独特的睡前一次给药时间表，这是对患者护理的重大贡献，使我们能够继续执行商业发布并扩大LUMRYZ在发作性睡病社区的影响力。”
关于用于缓释口服混悬液的 LUMRYZ（羟丁酸钠）
LUMRYZ 是一种缓释羟丁酸钠药物，于 2023 年 5 月 1 日获得 FDA 批准，是第一种也是唯一一种针对发作性睡病成人猝倒或日间过度嗜睡 (EDS) 的睡前治疗药物。2024 年 10 月 16 日，LUMRYZ 还获准作为 7 岁及以上发作性睡病儿科患者的一次睡前猝倒或 EDS 的睡前治疗药物。
美国食品药品管理局批准LUMRYZ得到了Rest-on结果的支持。REST-on是一项针对发作性睡病成人的随机、双盲、安慰剂对照的关键性3期试验。与安慰剂相比，LUMRYZ在三个共同主要终点：EDS（MWT）、临床医生对患者功能的总体评估（CGI-I）和瘫痪发作，显示出具有统计学意义且具有临床意义的改善。
美国食品药品管理局于2023年5月首次获得批准，授予LUMRYZ为期7年的孤儿药独家经营权，用于治疗成人发作性睡病的猝倒性或EDS，原因是发现LUMRYZ相对于目前可用的含氧疗法具有临床优势。特别是，美国食品药品管理局发现，与目前可用的每晚两次的含氧产品相比，LUMRYZ通过提供每晚一次的给药方案，避免夜间醒来服用第二剂，为患者护理做出了重大贡献。同样，随着2024年10月获得儿科批准，美国食品药品管理局又向LUMRYZ授予了为期7年的孤儿药独家经营权，用于治疗7岁及以上的发作性睡病患者的猝倒性或EDS，原因是发现LUMRYZ相对于目前可用的含氧疗法具有临床优势。特别是，美国食品和药物管理局在那里发现，LUMRYZ的剂量为最大限度地减少睡眠分散和睡眠结构的干扰提供了机会，而每晚服用两次羟丁酸给药方案的患者是不可能的，从而为患者护理做出了重大贡献。
关于 Avadel Pricals plc
Avadel Pharmaceuticals plc（纳斯达克股票代码：AVDL）是一家生物制药公司，专注于改造药物以改变生活。我们的方法包括应用创新的解决方案来开发药物，以应对患者在当前治疗方案中面临的挑战。Avadel 的商业产品 LUMRYZ 已被美国食品药品监督管理局 (FDA) 批准为第一款也是唯一一种用于治疗 7 岁及以上发作性睡病患者的猝倒或日间过度嗜睡 (EDS) 的睡前氧丁酸。欲了解更多信息，请访问。
重要的安全信息
警告：将LUMRYZ（羟丁酸钠）与其他中枢神经系统（CNS）抑制剂，例如用来让你入睡的药物，包括阿片类镇痛药、苯二氮卓类药物、镇静抗抑郁药、抗精神病药、镇静抗癫痫药物、全身麻醉剂、肌肉松弛剂、酒精或街头毒品，可能会导致严重的医疗问题，包括呼吸困难（呼吸抑制），低血压（低血压），警觉性变化（嗜睡），昏厥（晕厥）和死亡。
LUMRYZ（羟丁酸钠）的活性成分是伽玛羟丁酸盐（GHB）的一种形式，一种受控物质。单独或与其他中枢神经系统抑制剂（引起警觉或意识变化的药物）一起滥用或滥用非法的gHb会造成严重的副作用。这些影响包括癫痫发作、呼吸困难（呼吸抑制）、警觉性变化（嗜睡）、昏迷和死亡。如果您有任何这些严重的副作用，请立即致电您的医生。
由于这些风险，LUMRYZ只能通过处方获得，并通过LUMRYZ REMS的认证药房进行配送。您必须注册 LUMRYZ REMS 才能获得 LUMRYZ。欲了解更多信息，请致电1-877-453-1029或致电。 |
适应症
LUMRYZ（羟丁酸钠）用于缓释口服混悬液是一种处方药，用于治疗 7 岁及以上发作性睡病患者的以下症状：
突然出现肌肉无力或麻痹（瘫痪）
白天过度嗜睡 (EDS)
如果你正在服用或你的孩子服用其他睡眠药物或镇静剂（导致困倦的药物）、饮酒或有一种称为琥珀酸半醛脱氢酶缺乏症的罕见问题，请不要服用 LUMRYZ。
将 LUMRYZ 保存在安全的地方，以防止滥用和滥用。出售或赠送 LUMRYZ 可能会伤害他人，并且是违法的。如果您或您的孩子曾经滥用或依赖酒精、处方药或街头毒品，请告诉您的医生。
任何服用 LUMRYZ 的人在服用 LUMRYZ 后的至少六 (6) 小时内不应做任何需要完全清醒或有危险的事情，包括开车、使用重型机械或驾驶飞机。在你知道LUMRYZ如何影响你之前，不应进行这些活动。
快速入睡，包括站立时或从床上起床时，会导致跌倒并受伤，一些人需要住院。
LUMRYZ 可能会引起严重的副作用，包括：
呼吸问题，包括呼吸减慢、呼吸困难和/或睡觉时短暂不呼吸（例如睡眠呼吸暂停）。已经有呼吸或肺部问题的人服用 LUMRYZ 时出现呼吸问题的几率更高。
心理健康问题，包括精神错乱、看到或听到不真实的事物（幻觉）、不寻常或令人不安的想法（思维异常）、感到焦虑或沮丧、抑郁、自杀或试图自杀的想法、疲劳加剧、内疚感或一文不值以及难以集中注意力。如果您或您的孩子患有或患有抑郁症或曾试图伤害自己，请告诉您的医生。如果您或您的孩子出现心理健康问题症状或体重或食欲变化，请立即致电您的医生。
梦游。梦游会造成伤害。如果你或你的孩子开始梦游，请给你的医生打电话。
如果您或您的孩子限制盐分饮食或有高血压、心力衰竭或肾脏问题，请告诉您的医生。LUMRYZ 含有大量的钠（盐），可能不适合你。
LUMRYZ 在成人中最常见的副作用包括恶心、头晕、尿床、头痛和呕吐。当你服用更高剂量的LUMRYZ时，你的副作用可能会增加。儿童最常见的副作用包括恶心、尿床、呕吐、头痛、体重减轻、食欲下降、头晕和梦游。如果不按指示服用，LUMRYZ 可能会导致身体依赖和对药物的渴望。这些并不是 LUMRYZ 可能的全部副作用。
欲了解更多信息，请咨询您的医生或药剂师。致电您的医生，获取有关副作用的医疗建议。
我们鼓励您向 FDA 报告处方药的负面副作用。访问或致电 1-800-FDA-1088。
请查看完整的处方信息，包括 BOXED 警告。
