- 首次在淤胆性肝病中成功进行全球黄金标准儿科3期试验
- PEDFIC 1和2研究是Bylvay在美国和欧洲获得批准的基础
- 研究表明,Bylvay在瘙痒严重程度评分和血清胆汁酸水平方面有统计学意义的改善。
热门资讯> 正文
2022-07-06 20:35
BOSTON, July 06, 2022 (GLOBE NEWSWIRE) -- Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company developing novel bile acid modulators, today announced that The Lancet Gastroenterology & Hepatology published the results of the pivotal PEDFIC 1 trial, which evaluated Bylvay® (odevixibat) for the treatment of progressive familial intrahepatic cholestasis (PFIC). The study met both of its two primary endpoints, demonstrating that Bylvay provided statistically significant improvements in pruritus severity scores and reductions in serum bile acid levels, with rapid, sustained, and consistent results. The full manuscript of the PEDFIC 1 study is available on The Lancet Gastroenterology & Hepatology website.
波士顿,2022年7月6日(环球社)--纳斯达克公司(Albireo Pharma,Inc.),一家开发新型胆汁酸调节剂的罕见肝病公司,今天宣布,《柳叶刀》胃肠病学与肝脏病学公布了评估Bylvay的关键PEDFIC 1试验的结果®治疗进行性家族性肝内胆汁淤积症(PFIC)。这项研究同时满足了它的两个主要终点,表明Bylvay在瘙痒严重程度评分和血清胆汁酸水平方面都有统计上的显著改善,结果迅速、持续和一致。PEDFIC 1研究的完整手稿可在《柳叶刀》胃肠病和肝病学网站上找到。
A potent, once-daily, non-systemic ileal bile acid transporter inhibitor (IBATi), Bylvay has minimal systemic exposure and acts locally in the small intestine. Bylvay was recently approved in the U.S. for the treatment of pruritus in patients 3 months of age and older with all types of PFIC, and in Europe for the treatment of all types of PFIC in patients aged 6 months or older, based on the PEDFIC 1 study and PEDFIC 2 open-label extension (OLE) study.
作为一种有效的、每天一次的非全身性回肠胆汁酸转运蛋白抑制剂(IBATi),Bylvay具有最小的全身性暴露,并在小肠局部起作用。根据PEDFIC 1研究和PEDFIC 2开放标签扩展(OLE)研究,Bylvay最近在美国被批准用于治疗3个月及以上患有所有类型PFIC的患者的瘙痒,在欧洲被批准用于治疗6个月或以上患者的所有类型的PFIC。
"The PEDFIC 1 study is the largest trial ever completed in PFIC and we are pleased that a prestigious journal has recognized the quality of the science and importance of the landmark results," said Ron Cooper, President and Chief Executive Officer of Albireo. "The study outcomes have been positively received by physicians and payors enabling swift pricing and reimbursement reviews of Bylvay, helping to accelerate access to this much needed medicine for patients suffering with this cholestatic liver disease."
Albireo首席执行官罗恩·库珀、总裁说:“PEDFIC 1号研究是PFIC有史以来完成的最大规模的试验,我们很高兴一家著名的杂志承认了科学的质量和里程碑式结果的重要性。研究结果得到了医生和付款人的积极响应,使Bylvay能够迅速定价和报销审查,帮助患有这种淤胆性肝病的患者更快地获得这种急需的药物。“
The publication includes data from the randomized, double-blind, placebo-controlled Phase 3 PEDFIC 1 global study. Within the study findings:
该出版物包括来自随机、双盲、安慰剂对照的3PEDFIC 1期全球研究的数据。在研究结果中:
Patients with PFIC have impaired bile flow, or cholestasis, and the resulting bile build-up in liver cells causes liver disease and symptoms including intense itching, poor sleep, delayed growth and diminished quality of life. The harmful impacts of the disease extend to parents and caregivers, as the 2022 multinational PICTURE study revealed that PFIC negatively affects caregivers' quality of life, relationships, and career prospects.
PFIC患者的胆汁流动受损或胆汁淤积,由此导致的胆汁在肝细胞中积聚会导致肝脏疾病和症状,包括剧烈瘙痒、睡眠不佳、生长迟缓和生活质量下降。这种疾病的有害影响延伸到父母和照顾者,因为2022年多国图片研究显示,PFIC对照顾者的生活质量、关系和职业前景产生了负面影响。
The results from the PEDFIC studies provide confidence in the translatability of the data in Alagille syndrome (ALGS) and biliary atresia and the two Phase 3 studies in progress. The ASSERT study in ALGS is fully enrolled and topline data is expected by the end of the year. The BOLD study in biliary atresia is expected to complete enrollment this year with topline data available in 2024.
PEDFIC研究的结果为Alagille综合征(ALGS)和胆道闭锁以及正在进行的两项3期研究的数据的可译性提供了信心。ALGS中的ASSET研究已经完全登记在册,预计今年年底将公布背线数据。这项针对胆道闭锁的大胆研究预计将于今年完成,并在2024年提供背线数据。
About Bylvay (odevixibat)
关于Bylvay(奥德维西巴特)
Bylvay is the first drug approved in the U.S. for the treatment of pruritus in patients 3 months of age and older in all types of progressive familial intrahepatic cholestasis (PFIC). Limitation of Use: Bylvay may not be effective in PFIC type 2 patients with ABCB11 variants resulting in non-functional or complete absence of bile salt export pump protein (BSEP-3). The European Commission (EC) and UK Medicines and Healthcare Products Regulatory Agency (MHRA) have also granted marketing authorization of Bylvay for the treatment of PFIC in patients aged 6 months or older. Bylvay is available in Germany and the UK and will be available for sale in other European countries following pricing and reimbursement approval. A potent, once-daily, non-systemic ileal bile acid transporter inhibitor (IBATi), Bylvay has minimal systemic exposure and acts locally in the small intestine. Bylvay can be taken as a capsule for patients that are able to swallow capsules, or opened and sprinkled onto food, which is a factor of key importance for adherence in a pediatric patient population. The most common adverse reactions for Bylvay are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency. The medicine can only be obtained with a prescription. For more information about using Bylvay, see the package leaflet or contact your doctor or pharmacist. For full prescribing information, visit .
Bylvay是美国批准的第一种药物,用于治疗所有类型的进行性家族性肝内胆汁淤积症(PFIC)3个月及以上患者的瘙痒。使用限制:Bylvay可能对具有ABCB11变异的2型PFIC患者无效,导致无功能或完全缺乏胆盐输出泵蛋白(BSEP-3)。欧盟委员会(EC)和英国药品和保健产品监管机构(MHRA)也批准了Bylvay的营销授权,用于治疗6个月或以上患者的PFIC。Bylvay在德国和英国销售,在定价和报销批准后,将在其他欧洲国家销售。作为一种有效的、每天一次的非全身性回肠胆汁酸转运蛋白抑制剂(IBATi),Bylvay具有最小的全身性暴露,并在小肠局部起作用。Bylvay可以作为能够吞咽胶囊的患者的胶囊,或者打开并洒在食物上,这是在儿科患者群体中坚持的一个关键因素。Bylvay最常见的不良反应是腹泻、肝功能异常、呕吐、腹痛和脂溶性维生素缺乏。这种药只能凭处方才能拿到。有关使用Bylvay的更多信息,请参阅包装传单或联系您的医生或药剂师。有关完整的处方信息,请访问。
In the U.S. and Europe, Bylvay has orphan exclusivity for its approved PFIC indications, and orphan designations for the treatment of ALGS, biliary atresia and primary biliary cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2 open-label trial in patients with PFIC, in the BOLD Phase 3 study for patients with biliary atresia and the ASSERT Phase 3 study for ALGS.
在美国和欧洲,Bylvay因其批准的PFIC适应症而获得孤儿专有权,并因治疗ALGS、胆道闭锁和原发性胆管炎而被指定为孤儿。Bylvay正在进行的针对PFIC患者的PEDFIC 2开放标签试验、针对胆道闭锁患者的BOLD第三阶段研究以及针对ALGS的Assert第三阶段研究中进行评估。
Important Safety Information
重要安全信息
About Albireo
关于Albireo
Albireo Pharma is a rare disease company focused on the development of novel bile acid modulators to treat rare pediatric and adult liver diseases. Albireo's lead product, Bylvay, was approved by the U.S. FDA as the first drug for the treatment of pruritus in all types of progressive familial intrahepatic cholestasis (PFIC), and it is also being developed to treat other rare pediatric cholestatic liver diseases with Phase 3 trials in Alagille syndrome (ALGS) and biliary atresia, as well as Open-label Extension (OLE) studies for PFIC and ALGS. In Europe, Bylvay has been approved for the treatment of PFIC with pricing listing in Germany and guidance from the National Institute for Health and Care Excellence (NICE) recommending Bylvay for use in the National Health Service in England, Wales and Northern Ireland. The Company has also completed a Phase 1 clinical trial for A3907 to advance development in adult cholestatic liver disease, with IND-enabling studies progressing with A2342 for viral and cholestatic liver disease. Albireo was spun out from AstraZeneca in 2008 and is headquartered in Boston, Massachusetts, with its key operating subsidiary in Gothenburg, Sweden. For more information on Albireo, please visit .
Albireo Pharma是一家罕见疾病公司,专注于开发新型胆汁酸调节剂来治疗罕见的儿童和成人肝病。Albireo的主导产品Bylvay被美国FDA批准为第一种用于治疗所有类型进行性家族性肝内胆汁淤积症(PFIC)的瘙痒的药物,还在开发用于治疗其他罕见的儿童淤胆性肝病的药物,Alagille综合征(ALGS)和胆道闭锁的第三阶段试验,以及针对PFIC和ALGS的开放标签扩展(OLE)研究。在欧洲,Bylvay已被批准用于治疗PFIC,在德国上市定价,国家健康与护理卓越研究所(NICE)指导建议将Bylvay用于英格兰、威尔士和北爱尔兰的国家医疗服务。该公司还完成了A3907的第一阶段临床试验,以促进成人胆汁淤积性肝病的开发,A2342用于病毒性和胆汁淤积性肝病的IND研究取得进展。Albireo于2008年从阿斯利康剥离出来,总部位于马萨诸塞州波士顿,其主要运营子公司位于瑞典哥德堡。有关Albireo的更多信息,请访问。
Forward-Looking Statements
前瞻性陈述
This press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements, other than statements of historical fact, regarding, among other things: Albireo's commercialization plans; the plans for, or progress, scope, cost, initiation, duration, enrollment, results or timing for availability of results of, development of Bylvay or any other Albireo product candidate or program; the PEDFIC 2 open-label trial in patients with PFIC; the pivotal trial for Bylvay in biliary atresia (BOLD); the pivotal trial for Bylvay in Alagille syndrome (ASSERT);; the target indication(s) for development or approval; the timing for initiation or completion of or availability or reporting of results from any clinical trial, including the long-term open-label extension study for Bylvay in PFIC, the BOLD and ASSERT trials, potential regulatory approval and plans for potential commercialization of Bylvay in additional countries; the potential benefits or competitive position of Bylvay or any other Albireo product candidate or program or the commercial opportunity in any target indication; or Albireo's plans, expectations or future operations, financial position, revenues, costs or expenses. Albireo often uses words such as "anticipates," "believes," "plans," "expects," "projects," "future," "intends," "may," "will," "should," "could," "estimates," "predicts," "potential," "planned," "continue," "guidance," or the negative of these terms or other similar expressions to identify forward-looking statements. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to: results achieved in Bylvay in the treatment of patients with PFIC may be different than observed in clinical trials, and may vary among patients; potential negative impacts of the COVID-19 pandemic, including on manufacturing, supply, conduct or initiation of clinical trials, or other aspects of our business; whether favorable findings from clinical trials of Bylvay to date, including findings in indications other than PFIC, will be predictive of results from other clinical trials of Bylvay; the timing for initiation or completion of, or for availability of data from, clinical trials of Bylvay, including BOLD and ASSERT and the outcomes of such trials; Albireo's ability to obtain coverage, pricing or reimbursement for approved products in the United States or Europe; delays or other challenges in the recruitment of patients for, or the conduct of, the Company's clinical trials; and the Company's critical accounting policies. These and other risks and uncertainties that Albireo faces are described in greater detail under the heading "Risk Factors" in Albireo's most recent Annual Report on Form 10-K or in subsequent filings that it makes with the Securities and Exchange Commission. As a result of risks and uncertainties that Albireo faces, the results or events indicated by any forward-looking statement may not occur. Albireo cautions you not to place undue reliance on any forward-looking statement. In addition, any forward-looking statement in this press release represents Albireo's views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Albireo disclaims any obligation to update any forward-looking statement except as required by applicable law.
本新闻稿包括“1995年私人证券诉讼改革法”所指的“前瞻性陈述”。除历史事实的陈述外,前瞻性陈述包括但不限于:Albireo的商业化计划;Bylvay或任何其他Albireo候选产品或计划的开发、Bylvay或任何其他Albireo产品候选或计划的计划或进展、范围、成本、启动、持续时间、登记、结果或可获得结果的时间;针对PFIC患者的PEDFIC 2开放标签试验;Bylvay治疗胆道闭锁的关键试验(BOLD);Bylvay在Alagille综合征的关键试验(断言);开发或批准的目标适应症;任何临床试验的启动或完成时间、可获得性或结果报告,包括Bylvay在PFIC中的长期开放标签扩展研究、大胆和确凿的试验、潜在的监管批准和Bylvay在更多国家的潜在商业化计划;Bylvay或任何其他Albireo候选产品或计划的潜在好处或竞争地位,或任何目标适应症中的商业机会;或Albireo的计划、预期或未来运营、财务状况、收入、成本或支出。Albireo经常使用“预期”、“相信”、“计划”、“预期”、“项目”、“未来”、“打算”、“可能”、“将”、“应该”、“可能”、“估计”、“预测”、“潜在”、“计划”、“继续”、“指导”或这些术语的否定或其他类似表述来识别前瞻性陈述。由于各种风险、不确定因素和其他因素,实际结果、业绩或经验可能与任何前瞻性陈述所表达或暗示的结果大不相同,包括, 但不限于:Bylvay在治疗PFIC患者方面取得的结果可能与临床试验中观察到的结果不同,并且可能因患者而异;新冠肺炎大流行的潜在负面影响,包括对临床试验的制造、供应、进行或启动,或我们业务的其他方面;迄今为止Bylvay临床试验的有利结果,包括PFIC以外的其他适应症的结果,是否可以预测Bylvay的其他临床试验的结果;启动或完成Bylvay临床试验的时间,或获得Bylvay临床试验数据的时间,包括Bylvay临床试验的大胆和可靠,以及此类试验的结果;Albireo在美国或欧洲获得批准产品的承保范围、定价或报销的能力;在招募患者或进行公司临床试验方面的延迟或其他挑战;以及公司的关键会计政策。Albireo面临的这些和其他风险和不确定因素在Albireo公司最新的10-K表格年度报告或其提交给证券交易委员会的后续文件中的“风险因素”标题下有更详细的描述。由于Albireo公司面临的风险和不确定因素,任何前瞻性陈述所表明的结果或事件可能不会发生。Albireo告诫您不要过度依赖任何前瞻性声明。此外,本新闻稿中的任何前瞻性陈述仅代表Albireo截至本新闻稿发布之日的观点,不应被视为代表其在任何后续日期的观点。除非适用法律要求,否则Albireo不承担更新任何前瞻性陈述的义务。
Media Contact:
Colleen Alabiso, 857-356-3905, colleen.alabiso@albireopharma.com
Lance Buckley, 917-439-2241, lbuckley@lippetaylor.com
媒体联系人:
Colleen Alabiso电话:857-356-3905电子邮件:colleen.alabiso@albireophma.com
兰斯·巴克利,电话:917-439-2241,电子邮件:luckley@lippetaylor.com
Investor Contact:
Hans Vitzthum, LifeSci Advisors, LLC., 617-430-7578
投资者联系方式:
Hans Vitzthum,LifeSci Advisors,LLC,617-430-7578