Stiripentol

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。 12月20日，据CDE官网最新公示，石家庄四药和科伦药业提交的4类仿制药甲磺酸沙非胺片上市申请获得受理，该品种属于一种治疗帕金森病的药物。据摩熵医药数据库显示，该品种目前国内暂无仿制药企获批上市，若石家庄四药和科伦药业能顺利获批，有望拿下国内首仿。 截图来源：CDE 帕金森病（PD）是一种神经系统退行性疾病，主要表现为运动迟缓、静止性震颤、肌肉僵直及姿势平衡障碍等，其发病是由于多巴胺神经系统退化导致大脑中神经递质多巴胺缺乏。PD主要影响中老年人，随着年龄的增长，患病率逐渐增高。公开数据显示，我国65岁以上老年人的PD患病率为1.7%，据此推算我国PD患者数量或超300万。预计随着人口老龄化的加剧，我国可能成为全球PD患者人口最多的国家。 抗PD市场持续增长，两家药企猛攻百亿蓝海 根据百谏方略（DIResaerch）研究统计，全球帕金森病药物市场规模呈现稳步扩张的态势，2023年全球帕金森病药物市场销售额为382亿元，预计2030年将达到612.4亿元，2023-2030期间年复合增长率（CAGR）为6.97%。根据摩熵医药数据显示，2022年中国帕金森化药市场近30亿元，其中医院端23.13亿元，零售端5.99亿元。 2018-2022年全国院内帕金森药物市场规模数据来源：摩熵医药全国医院销售（全终端）数据库 2018-2022年全国零售帕金森药物市场规模数据来源：摩熵医药全国药店零售数据库 甲磺酸沙非胺由意大利制药公司Newron原研，是一款选择性抑制B型单胺氧化酶（MAO-B）抑制剂。自2015年2月在欧盟获批上市以来，甲磺酸沙非胺凭借其独特的作用机制，在治疗接受稳定剂量左旋多巴治疗的、出现运动波动的特发性帕金森病方面表现出色。2017年3月，甲磺酸沙非胺片获FDA批准上市，进一步巩固了其在全球帕金森病治疗领域的地位。 截图来源：摩熵医药（原药融云）中国药品批文数据库 在国内市场，甲磺酸沙非胺的独家经营权目前由Zambon SpA（赞邦）公司持有。然而，随着石家庄四药和科伦药业对甲磺酸沙非胺仿制药的研发进展，国内抗帕金森病药物市场即将迎来新的竞争格局。若两家药企能顺利获批，不仅将夺得国内首仿的地位，更将为规模超过百亿的国内抗帕金森病药物市场注入新的动力，推动市场规模的进一步扩张。 石四药深耕多个疾病领域，拿下超5千万帕金森药国内首仿 石家庄四药集团专注于抗感染、呼吸系统、神经系统、心脑血管以及抗肿瘤等疾病领域，已构建起一条全面且丰富的产品线，涵盖已上市及在研药物。在神经系统疾病领域，石四药尤为突出，拥有多款通过评价的药物，如首家过评的布南色林片、司替戊醇干混悬剂，以及前三家过评的对乙酰氨基酚甘露醇注射液和盐酸罗哌卡因注射液等。 今年6月28日，石家庄四药的卡左双多巴缓释片获得NMPA批准上市，为该品种国内首仿。卡左双多巴缓释片是一款复方抗帕金森药，据摩熵医药数据库显示，该品种在2023年全国医院(全终端)销售额超5千万，同比增长达20.30%。 截图来源：摩熵医药（原药融云）全国医院销售(全终端)数据库 此外，在国内市场，还有精华制药、重庆华邦制药、浙江华海药业、广州绿十字制药等7家企业提交了卡左双多巴缓释片的上市申请，市场竞争激烈。 结语 面对帕金森病这一严重影响中老年人生活质量的疾病，以及不断增长的市场规模，石家庄四药与科伦药业在甲磺酸沙非胺片仿制药领域的竞争无疑将为患者提供更多的治疗选择。在这场百亿蓝海的争夺战中，谁将最终脱颖而出，让我们共同期待。 END 本文为原创文章，转载请留言获取授权  近期热门资源获取 后台回复关键词“深度报告”，获取价值18600元，报告大礼包 后台回复关键词“2023首仿”，获取2023年首仿药物获批名单 后台回复“GLP-1深度报告”，获取完整《GLP-1产业现状与未来发展》PDF版。 后台回复“中国 I 类新药”，获取完整《中国 I 类新药靶点》PDF版。 后台回复“NAFLD/NASH”，获取完整《NAFLD/NASH报告》PDF版。 后台回复“AI+制药”，获取完整《中国AI制药企业白皮书》PDF版。 后台回复“XXG”，获取完整《中国心血管系统药物分析报告》PDF版。 后台回复“FZZJ”，获取完整《基于剂型改良的复杂注射剂分析》PDF版。 后台回复“药品进出口”，获取完整《中国药品进出口白皮书》深度报告-PDF版。 联系我们，体验摩熵医药更多专业服务 会议 合作 园区 服务 数据库 咨询 定制 服务 媒体 合作 👆👆👆点击上方图片，即可开启摩熵化学数据查询 点击阅读原文，申请摩熵医药企业版免费试用！

Data from nine abstracts to be presented, including first data from the EpiCom Trial, a prospective evaluation of behavioral outcomes in patients with tuberous sclerosis complex, which suggests improvements in severity of behavioral symptoms Real-world data from the BECOME (BEhavior, COgnition and More with Epidiolex®) surveys, which show outcomes reported by long-term care facility nurses and tuberous sclerosis complex caregivers For U.S. media and investors only DUBLIN, Dec. 6, 2024 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced nine company-sponsored Epidiolex® (cannabidiol) posters are being presented at the American Epilepsy Society (AES) 2024 Annual Meeting, being held December 6-10 in Los Angeles, California. Data presented at the meeting includes updated analyses of real-world data from the BECOME-TSC (BEhavior, COgnition, and More with Epidiolex) caregiver survey, which characterizes and quantifies seizure and non-seizure outcomes in patients with epilepsy and tuberous sclerosis complex (TSC) treated with Epidiolex. An additional presentation showcases data from the BECOME-LTC survey, which evaluated the perspectives of nurses who care for patients with epilepsy in long-term care (LTC) facilities and group homes, reporting improvements in seizure frequency and in certain non-seizure outcomes associated with Epidiolex treatment. Further, the first presentation of data from the EpiCom trial, a prospective, interventional trial evaluating the impact of adjunctive Epidiolex on TSC-associated neuropsychiatric disorders (TAND), revealed improvements in behavioral symptom severity following treatment initiation. "Our real-world data presentations at AES 2024, including novel findings from the BECOME-LTC, BECOME-TSC and EpiCom studies, demonstrate the meaningful impact of Epidiolex in the treatment of patients with rare epilepsies," said Sarah Akerman, MD, head of neuroscience global medical and scientific affairs of Jazz Pharmaceuticals. "These findings increase our understanding of Epidiolex's benefits beyond seizure control, addressing unmet needs across a range of epilepsy syndromes for people living with rare epilepsies and demonstrating reproducibility and consistency of effect across different populations." Data highlights include: A prespecified three-month analysis of the EpiCom trial reporting TSC-associated neuropsychiatric disorders (TAND)-associated outcomes demonstrated improvements after initiating adjunctive Epidiolex treatment in the severity of behavioral problems in patients with TSC as reported by the TAND Self-Report Quantified Checklist and Aberrant Behavior Checklist. Two updated analyses of real-world outcomes from the BECOME-TSC study showed that, of 55 caregivers who completed the survey, 89% planned to continue Epidiolex treatment for their loved one. The most important stated reasons for continuing Epidiolex included seizure and non-seizure benefits such as reduced seizure frequency and severity/duration, as well as TAND-related improvements in cognition and language/communication. Results from the BECOME-LTC (BEhavior, COgnition, and More with Epidiolex in the Long-Term Care Setting) survey found that, among 102 nurses surveyed, 85% reported a reduction in overall frequency of any seizure type after Epidiolex initiation, with 49% reporting a greater than 50% reduction. Improvements were also observed across different seizure subtypes as well as in non-seizure outcomes, with nurses reporting improvements in emotional functioning, sleep, cognitive abilities, ability to communicate, and physical functioning. Results from CARE-EpiC (Caregiver Analysis of Real-world Epidiolex in Epilepsy Context), a cross-sectional caregiver survey, demonstrated reduced caregivers' need for additional support of their dependents' physical, emotional, and behavioral care after Epidiolex initiation and characterized improvements in their dependents' well-being as well as caregivers' experiences. A subgroup analysis evaluating treatment outcomes in patients with TSC (TSC group) versus other types of focal epilepsy (non-TSC group) treated with Epidiolex in the U.S. Expanded Access Program, found Epidiolex has similar effectiveness in TSC and other focal epilepsies, regardless of focal epilepsy type, further reinforcing the clinical profile of Epidiolex as a broad-spectrum agent. In the TSC group, Epidiolex was associated with a median reduction from baseline of 51%–87% in focal seizures and 44%–87% in total seizures. In the non-TSC group, Epidiolex was also associated with a median reduction from baseline of 46%–75% in focal and 46%–74% in total seizures. Epidiolex is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or TSC in patients one year of age and older. All AES 2024 abstracts are available online at the following link: . A full list of Jazz Pharmaceuticals' presentations follows below: About Tuberous Sclerosis Complex Tuberous sclerosis complex (TSC) is a rare genetic condition.1 The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs2 and is a leading cause of genetic epilepsy.3 People with TSC may experience a variety of seizure types. One of the most common is infantile spasms that typically present in the first year of life; focal (or partial) seizures are also very common.4 TSC is associated with an increased risk of autism and intellectual disability5 and the severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.4 Epilepsy is present in about 85 percent of patients with TSC and may progress to become intractable to medication.4,6,7 More than 60 percent of individuals with TSC do not achieve seizure control8 with standard treatments such as antiepileptic drugs, epilepsy surgery, ketogenic diet, or vagus nerve stimulation8 compared to 30-40 percent of individuals with epilepsy who do not have TSC who are drug resistant.9,10 About Dravet Syndrome Dravet syndrome (DS) is a rare genetic condition that appears during the first year of life with frequent fever-related seizures (febrile seizures). Later, other types of seizures typically arise, including myoclonic seizures (involuntary muscle spasms).11 Additionally, status epilepticus, a potentially life-threatening state of continuous seizure activity requiring emergency medical care, may occur. Children with DS typically experience poor development of language and motor skills, hyperactivity and difficulty relating to others. About Lennox-Gastaut Syndrome Lennox-Gastaut syndrome (LGS) begins in childhood. It is characterized by multiple types of seizures. People with LGS begin having frequent seizures in early childhood, usually between ages 3 and 5.12 More than three-quarters of affected individuals have tonic seizures, which cause the muscles to contract uncontrollably. Almost all children with LGS develop learning problems and intellectual disability. Many also have delayed development of motor skills such as sitting and crawling. Most people with LGS require help with usual activities of daily living. About Epidiolex®/Epidyolex® (cannabidiol) Epidiolex/Epidyolex is a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD). Cannabidiol, the active ingredient in Epidiolex, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant. The precise mechanisms by which Epidiolex exerts its anticonvulsant effect in humans are unknown. Epidiolex was approved by the U.S. Food and Drug Administration (FDA) for use in the U.S., the European Commission (EC) for use in Europe, the Medicines and Healthcare products Regulatory Agency (MHRA) for use in Great Britain, the Therapeutic Goods Administration for use in Australia, Swissmedic for use in Switzerland, the Food & Nutrition Services of the Israel Ministry of Health for use in Israel, and the New Zealand Medicines and Medical Devices Safety Authority for use in New Zealand, is an oral solution which contains highly purified cannabidiol (CBD). In the U.S., Epidiolex is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex (TSC) in patients one year of age and older. Epidiolex has received approval in the European Union under the tradename Epidyolex for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and DS in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older. Epidiolex has received Orphan Drug Designation (ODD) from the U.S. FDA for the treatment of seizures associated with LGS, DS, and TSC. Similarly, Epidyolex received ODD from the European Medicines Agency (EMA) for the same indications. Important Safety Information & Indications CONTRAINDICATION: HYPERSENSITIVITY EPIDIOLEX (cannabidiol) oral solution is contraindicated in patients with a history of hypersensitivity to cannabidiol or any ingredients in the product. WARNINGS & PRECAUTIONS Hepatic Injury: EPIDIOLEX can cause dose-related transaminase elevations. Concomitant use of valproate and elevated transaminase levels at baseline increase this risk. Obtain transaminase and bilirubin levels prior to starting treatment, at 1, 3, and 6 months after initiation of treatment, and periodically thereafter, or as clinically indicated. Resolution of transaminase elevations occurred with discontinuation of EPIDIOLEX, reduction of EPIDIOLEX and/or concomitant valproate, or without dose reduction. For patients with elevated transaminase levels, consider dose reduction or discontinuation of EPIDIOLEX or concomitant medications known to affect the liver (e.g., valproate or clobazam). Dose adjustment and slower dose titration is recommended in patients with moderate or severe hepatic impairment. Consider not initiating EPIDIOLEX in patients with evidence of significant liver injury. There have been postmarketing reports of cholestatic or mixed patterns of liver injury. Elevated ammonia levels were reported in some patients with transaminase elevations; most taking concomitant valproate, clobazam, or both. Consider discontinuation or dose adjustment of valproate or clobazam if ammonia is elevated. Somnolence and Sedation: EPIDIOLEX can cause somnolence and sedation that generally occurs early in treatment and may diminish over time; these effects occur more commonly in patients using clobazam and may be potentiated by other CNS depressants. Suicidal Behavior and Ideation: Antiepileptic drugs (AEDs), including EPIDIOLEX, increase the risk of suicidal thoughts or behavior. Inform patients, caregivers, and families of the risk and advise them to monitor and report any signs of depression, suicidal thoughts or behavior, or unusual changes in mood or behavior. If these symptoms occur, consider if they are related to the AED or the underlying illness. Withdrawal of Antiepileptic Drugs: As with most AEDs, EPIDIOLEX should generally be withdrawn gradually because of the risk of increased seizure frequency and status epilepticus. ADVERSE REACTIONS: The most common adverse reactions in patients receiving EPIDIOLEX (≥10% and greater than placebo) include transaminase elevations; somnolence; decreased appetite; diarrhea; pyrexia; vomiting; fatigue, malaise, and asthenia; rash; insomnia, sleep disorder and poor-quality sleep; and infections. Hematologic abnormalities were also observed. PREGNANCY: EPIDIOLEX should be used during pregnancy only if the potential benefit justifies the potential risk to the fetus. Encourage women who are taking EPIDIOLEX during pregnancy to enroll in the EPIDIOLEX Pregnancy Surveillance Program and the North American Antiepileptic Drug (NAAED) Pregnancy Registry. DRUG INTERACTIONS: Strong inducers of CYP3A4 and CYP2C19 may affect EPIDIOLEX exposure. EPIDIOLEX may affect exposure to CYP2C19 substrates (e.g., clobazam, diazepam, stiripentol), orally administered P-gp substrates, or other substrates (see full Prescribing Information). Consider dose reduction of orally administered everolimus, with appropriate therapeutic drug monitoring, when everolimus is combined with EPIDIOLEX. A lower starting dose of everolimus is recommended when added to EPIDIOLEX therapy. Concomitant use of EPIDIOLEX and valproate increases the incidence of liver enzyme elevations. Pneumonia was observed more frequently with concomitant use of EPIDIOLEX and clobazam. Dosage adjustment of EPIDIOLEX or other concomitant medications may be necessary. INDICATIONS: EPIDIOLEX (cannabidiol) oral solution is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in patients 1 year of age and older. Please read the EPIDIOLEX full Prescribing Information for additional important information here . About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Contacts: Media: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 Investors: Jeff Macdonald Executive Director, Investor Relations Jazz Pharmaceuticals plc [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 SOURCE Jazz Pharmaceuticals plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED