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）——專注於改造藥物以改變生活的生物製藥公司Avadel Pharmicals plc（納斯達克股票代碼：AVDL）昨天宣佈，美國哥倫比亞特區地方法院（「法院」）在爵士製藥公司（「Jazz」）根據《行政訴訟法》提起的訴訟中作出了有利於美國食品藥品監督管理局（「FDA」）的裁決美國食品和藥物管理局批准了LumryzTM，這是第一種也是唯一一種針對成人猝倒或日間過度嗜睡的睡前氧氣療法發作性睡病。該裁決維持了對LUMRYZ的批准，因爲美國食品藥品管理局認定LUMRYZ在臨床上優於Jazz每晚兩次的含氧產品。
繼美國食品藥品管理局於2023年5月最終批准並授予LUMRYZ孤兒藥獨家經營權（「ODE」）之後，Jazz對美國食品藥品管理局提起訴訟，聲稱LUMRYZ的批准不符合《孤兒藥法》。Avadel CNS Pharmicals, LLC（「Avadel CNS」）進行了干預，爲美國食品藥品管理局的行爲辯護，併成功地主張維持對LUMRYZ的批准。
「我們對法院的裁決感到滿意，該裁決支持美國食品藥品管理局對LUMRYZ的臨床優勢裁決，同時最終批准用於發作性睡病成人。根據法院的裁決，LUMRYZ將繼續向發作性睡病界開放，並保留其孤兒藥獨家經營權。」 Avadel Pharmicals首席執行官格雷格·迪維斯說。「昨天的裁決進一步鞏固了LUMRYZ獨特的睡前一次給藥時間表，這是對患者護理的重大貢獻，使我們能夠繼續執行商業發佈並擴大LUMRYZ在發作性睡病社區的影響力。」
關於用於緩釋口服混懸液的 LUMRYZ（羥丁酸鈉）
LUMRYZ 是一種緩釋羥丁酸鈉藥物，於 2023 年 5 月 1 日獲得 FDA 批准，是第一種也是唯一一種針對發作性睡病成人猝倒或日間過度嗜睡 (EDS) 的睡前治療藥物。2024 年 10 月 16 日，LUMRYZ 還獲准作爲 7 歲及以上發作性睡病兒科患者的一次睡前猝倒或 EDS 的睡前治療藥物。
美國食品藥品管理局批准LUMRYZ得到了Rest-on結果的支持。REST-on是一項針對發作性睡病成人的隨機、雙盲、安慰劑對照的關鍵性3期試驗。與安慰劑相比，LUMRYZ在三個共同主要終點：EDS（MWT）、臨床醫生對患者功能的總體評估（CGI-I）和癱瘓發作，顯示出具有統計學意義且具有臨床意義的改善。
美國食品藥品管理局於2023年5月首次獲得批准，授予LUMRYZ爲期7年的孤兒藥獨家經營權，用於治療成人發作性睡病的猝倒性或EDS，原因是發現LUMRYZ相對於目前可用的含氧療法具有臨床優勢。特別是，美國食品藥品管理局發現，與目前可用的每晚兩次的含氧產品相比，LUMRYZ通過提供每晚一次的給藥方案，避免夜間醒來服用第二劑，爲患者護理做出了重大貢獻。同樣，隨着2024年10月獲得兒科批准，美國食品藥品管理局又向LUMRYZ授予了爲期7年的孤兒藥獨家經營權，用於治療7歲及以上的發作性睡病患者的猝倒性或EDS，原因是發現LUMRYZ相對於目前可用的含氧療法具有臨床優勢。特別是，美國食品和藥物管理局在那裏發現，LUMRYZ的劑量爲最大限度地減少睡眠分散和睡眠結構的干擾提供了機會，而每晚服用兩次羥丁酸給藥方案的患者是不可能的，從而爲患者護理做出了重大貢獻。
關於 Avadel Pricals plc
Avadel Pharmaceuticals plc（納斯達克股票代碼：AVDL）是一家生物製藥公司，專注於改造藥物以改變生活。我們的方法包括應用創新的解決方案來開發藥物，以應對患者在當前治療方案中面臨的挑戰。Avadel 的商業產品 LUMRYZ 已被美國食品藥品監督管理局 (FDA) 批准爲第一款也是唯一一種用於治療 7 歲及以上發作性睡病患者的猝倒或日間過度嗜睡 (EDS) 的睡前氧丁酸。欲了解更多信息，請訪問。
重要的安全信息
警告：將LUMRYZ（羥丁酸鈉）與其他中樞神經系統（CNS）抑制劑，例如用來讓你入睡的藥物，包括阿片類鎮痛藥、苯二氮卓類藥物、鎮靜抗抑鬱藥、抗精神病藥、鎮靜抗癲癇藥物、全身麻醉劑、肌肉鬆弛劑、酒精或街頭毒品，可能會導致嚴重的醫療問題，包括呼吸困難（呼吸抑制），低血壓（低血壓），警覺性變化（嗜睡），昏厥（暈厥）和死亡。
LUMRYZ（羥丁酸鈉）的活性成分是伽瑪羥丁酸鹽（GHB）的一種形式，一種受控物質。單獨或與其他中樞神經系統抑制劑（引起警覺或意識變化的藥物）一起濫用或濫用非法的gHb會造成嚴重的副作用。這些影響包括癲癇發作、呼吸困難（呼吸抑制）、警覺性變化（嗜睡）、昏迷和死亡。如果您有任何這些嚴重的副作用，請立即致電您的醫生。
由於這些風險，LUMRYZ只能通過處方獲得，並通過LUMRYZ REMS的認證藥房進行配送。您必須註冊 LUMRYZ REMS 才能獲得 LUMRYZ。欲了解更多信息，請致電1-877-453-1029或致電。 |
適應症
LUMRYZ（羥丁酸鈉）用於緩釋口服混懸液是一種處方藥，用於治療 7 歲及以上發作性睡病患者的以下症狀：
突然出現肌肉無力或麻痹（癱瘓）
白天過度嗜睡 (EDS)
如果你正在服用或你的孩子服用其他睡眠藥物或鎮靜劑（導致睏倦的藥物）、飲酒或有一種稱爲琥珀酸半醛脫氫酶缺乏症的罕見問題，請不要服用 LUMRYZ。
將 LUMRYZ 保存在安全的地方，以防止濫用和濫用。出售或贈送 LUMRYZ 可能會傷害他人，並且是違法的。如果您或您的孩子曾經濫用或依賴酒精、處方藥或街頭毒品，請告訴您的醫生。
任何服用 LUMRYZ 的人在服用 LUMRYZ 後的至少六 (6) 小時內不應做任何需要完全清醒或有危險的事情，包括開車、使用重型機械或駕駛飛機。在你知道LUMRYZ如何影響你之前，不應進行這些活動。
快速入睡，包括站立時或從牀上起牀時，會導致跌倒並受傷，一些人需要住院。
LUMRYZ 可能會引起嚴重的副作用，包括：
呼吸問題，包括呼吸減慢、呼吸困難和/或睡覺時短暫不呼吸（例如睡眠呼吸暫停）。已經有呼吸或肺部問題的人服用 LUMRYZ 時出現呼吸問題的幾率更高。
心理健康問題，包括精神錯亂、看到或聽到不真實的事物（幻覺）、不尋常或令人不安的想法（思維異常）、感到焦慮或沮喪、抑鬱、自殺或試圖自殺的想法、疲勞加劇、內疚感或一文不值以及難以集中注意力。如果您或您的孩子患有或患有抑鬱症或曾試圖傷害自己，請告訴您的醫生。如果您或您的孩子出現心理健康問題症狀或體重或食慾變化，請立即致電您的醫生。
夢遊。夢遊會造成傷害。如果你或你的孩子開始夢遊，請給你的醫生打電話。
如果您或您的孩子限制鹽分飲食或有高血壓、心力衰竭或腎臟問題，請告訴您的醫生。LUMRYZ 含有大量的鈉（鹽），可能不適合你。
LUMRYZ 在成人中最常見的副作用包括噁心、頭暈、尿牀、頭痛和嘔吐。當你服用更高劑量的LUMRYZ時，你的副作用可能會增加。兒童最常見的副作用包括噁心、尿牀、嘔吐、頭痛、體重減輕、食慾下降、頭暈和夢遊。如果不按指示服用，LUMRYZ 可能會導致身體依賴和對藥物的渴望。這些並不是 LUMRYZ 可能的全部副作用。
欲了解更多信息，請諮詢您的醫生或藥劑師。致電您的醫生，獲取有關副作用的醫療建議。
我們鼓勵您向 FDA 報告處方藥的負面副作用。訪問或致電 1-800-FDA-1088。
請查看完整的處方信息，包括 BOXED 警告。
