FDA Approves Medexus's Supplemental Biologics License Application for IXINITY(R) to Treat Hemophilia B in Pediatric Patients
FDA Approves Medexus's Supplemental Biologics License Application for IXINITY(R) to Treat Hemophilia B in Pediatric Patients
Expanded indication includes patients under 12 years of age, based on Phase 3/4 data demonstrating safety and efficacy in previously treated patients in this age group
根据3/4期数据,扩展的适应症包括12岁以下的患者,该年龄组先前接受过治疗的患者具有安全性和有效性
Toronto, Ontario and Chicago, Illinois--(Newsfile Corp. - March 26, 2024) - Medexus Pharmaceuticals (TSX: MDP) (OTCQX: MEDXF) today announced that the US Food and Drug Administration (FDA) recently approved Medexus's supplemental Biologics License Application (sBLA) for IXINITY [coagulation factor IX (recombinant)] for the on-demand, prophylactic, and perioperative treatment of pediatric patients under 12 years of age with hemophilia B. IXINITY, an intravenous recombinant factor IX therapeutic, is now approved for use in all patients with hemophilia B, a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood.
安大略省多伦多和伊利诺伊州芝加哥--(Newsfile Corp.,2024年3月26日)——Medexus Pharmicals(多伦多证券交易所股票代码:MDP)(OTCQX:MEDXF)今天宣布,美国食品药品监督管理局(FDA)最近批准了Medexus的IXINITY [凝血因子IX(重组)] 补充生物制剂许可申请(SbLa),用于按需、预防和周期对12岁以下儿童血友病患者进行手术治疗 B. IXINITY 是一种静脉注射重组 IX 因子治疗药物,现已获准用于所有 B 型血友病 a 患者以血液中缺乏凝血因子 IX 为特征的遗传性出血性疾病。
"We are pleased to secure approval for IXINITY for use in pediatric patients, a population that comprises approximately one third of individuals with hemophilia B in the United States*," said Ken d'Entremont, Chief Executive Officer of Medexus. "The newly expanded indication makes IXNITY a viable factor IX option for children living with hemophilia B. We hope the greater availability of IXINITY will help ease the burden on children and families who face the challenge of managing hemophilia B and allow children living with this lifelong condition to enjoy more active lives."
Medexus首席执行官肯·德恩特蒙特说:“我们很高兴获得IXINITY的批准,用于儿科患者,该人群约占美国乙型血友病患者的三分之一*。”“新扩大的适应症使IXINITY成为乙型血友病儿童的可行IXINITY选择。我们希望IXINITY的更多可用性将有助于减轻面临乙型血友病管理挑战的儿童和家庭的负担,并让终身患有这种疾病的儿童享受更积极的生活。”
"IXINITY effectively prevented and controlled bleeding episodes in the Phase 3/4 pediatric study," commented Prof Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, one of the study's principal investigators. "The study supported the efficacy and safety of IXINITY in pediatric patients, and the pharmacokinetics and safety profile were consistent with those observed in adults, although dose adjustment may be needed in pediatric patients. The results thus provide further validation of the clinical utility of IXINITY as a treatment for all people living with hemophilia B."
该研究的主要研究者之一、理学学士、mBBCH、MMed、FCPath的约翰尼·马朗古教授评论说:“在3/4期儿科研究中,IXINITY有效地预防和控制了出血发作。”“该研究支持了IXINITY对儿科患者的疗效和安全性,尽管儿科患者可能需要调整剂量,但其药代动力学和安全性特征与在成人中观察到的药代动力学和安全性一致。因此,研究结果进一步验证了IXINITY作为所有乙型血友病患者治疗的临床效用。”
The sBLA and expanded indication for IXINITY are based on results from a Phase 3/4 study that evaluated the pharmacokinetics (PK), safety, and efficacy of IXINITY as a prophylactic treatment in previously treated pediatric patients under 12 years of age with severe or moderately severe hemophilia B. The study demonstrated that prophylaxis with IXINITY was associated with low annualized bleeding rates, effective control of bleeding episodes, consistent PK, and a consistent safety profile. For more information about the study's methods, results, and conclusions, see "About the study" in Medexus's August 17, 2023 press release, available on the Investors-News & Events section of Medexus's corporate website.
IXINITY 的 sBLa 和扩展适应证基于一项3/4期研究的结果,该研究评估了IXINITY作为预防性治疗的药代动力学(PK)、安全性和有效性,用于先前接受过治疗的重度或中度血友病 B 的儿科患者。该研究表明,使用IXINITY进行预防与低年化出血率、有效控制出血发作、持续的PK有关,以及一致的安全概况。有关该研究的方法、结果和结论的更多信息,请参阅Medexus2023年8月17日新闻稿中的 “关于该研究”,该新闻稿可在Medexus公司网站的 “投资者新闻与活动” 部分获得。
* Source: World Federation of Hemophilia Report on the Annual Global Survey 2021; (accessed March 7, 2024).
*来源:世界血友病联合会2021年年度全球调查报告;(2024年3月7日访问)。
About IXINITY
关于 IXINITY
IXINITY [coagulation factor IX (recombinant)] Lyophilized Powder for Solution for Intravenous Injection is a coagulation factor IX (recombinant) indicated in adults and children with hemophilia B for on-demand treatment and control of bleeding episodes, perioperative management, and for routine prophylaxis to reduce the frequency of bleeding episodes.
IXINITY [凝血因子 IX(重组)] 用于静脉注射溶液的冻干粉末是一种凝血因子 IX(重组),适用于患有 B 型血友病的成人和儿童,用于按需治疗和控制出血发作、围手术期管理和常规预防以减少出血发作频率。
The most common adverse reaction observed in >2% of patients in clinical trials was headache. For more information about IXINITY, including important safety information, see the full prescribing information, which is available on the product's website at: .
在临床试验中,> 2% 的患者中观察到的最常见的不良反应是头痛。有关IXINITY的更多信息,包括重要的安全信息,请参阅完整的处方信息,该信息可在该产品的网站上获得,网址为:。
IXINITY is approved by the FDA for sale and use in the United States only and is not intended for export outside the United States. Medexus makes no representation that IXINITY is appropriate for, or authorized for sale to or use by, persons who are not located in the United States.
IXINITY 获得 FDA 的批准,仅可在美国销售和使用,不适用于美国境外出口。Medexus 未就IXINITY适用于不在美国的人员或授权其出售或由其使用作出任何陈述。
About hemophilia B
关于乙型血友病
Hemophilia B is a genetic bleeding disorder caused by a deficiency of coagulation factor IX, a protein needed to produce blood clots to stop bleeding. The clinical spectrum may include spontaneous or trauma-induced bleeding into joints, muscles, and soft tissues, resulting in joint damage, reduction in mobility, and severe arthritis, all of which negatively impact health-related quality of life. The primary aim of care is to prevent and treat bleeding by replacing the deficient clotting factor.
B型血友病是一种遗传性出血性疾病,由凝血因子IX缺乏引起,凝血因子IX是一种产生血块以止血所需的蛋白质。临床范围可能包括自发性或创伤引起的关节、肌肉和软组织出血,导致关节损伤、活动能力下降和严重关节炎,所有这些都会对与健康相关的生活质量产生负面影响。护理的主要目的是通过替换缺乏的凝血因子来预防和治疗出血。
About Medexus
关于 Medexus
Medexus is a leading specialty pharmaceutical company with a strong North American commercial platform and a growing portfolio of innovative and rare disease treatment solutions. Medexus's current focus is on the therapeutic areas of oncology, hematology, rheumatology, auto-immune diseases, allergy, and dermatology. For more information about Medexus and its product portfolio, please see the company's corporate website at and its filings on SEDAR+ at .
Medexus是一家领先的特种制药公司,拥有强大的北美商业平台以及不断增长的创新和罕见病治疗解决方案组合。Medexus目前的重点是肿瘤学、血液学、风湿病、自身免疫性疾病、过敏和皮肤病学等治疗领域。有关Medexus及其产品组合的更多信息,请访问该公司的公司网站及其在SEDAR+上的文件,网址为。
Contacts
联系人
Ken d'Entremont | CEO, Medexus Pharmaceuticals
Tel: 905-676-0003 | Email: ken.dentremont@medexus.com
Ken d'entremont | Medexus 制药首席执行官
电话:905-676-0003 | 电子邮件:ken.dentremont@medexus.com
Marcel Konrad | CFO, Medexus Pharmaceuticals
Tel: 312-548-3139 | Email: marcel.konrad@medexus.com
马塞尔·康拉德 | Medexus 制药首席财务官
电话:312-548-3139 | 电子邮件:marcel.konrad@medexus.com
Victoria Rutherford | Adelaide Capital
Tel: 480-625-5772 | Email: victoria@adcap.ca
维多利亚·卢瑟福 | 阿德莱德资本
电话:480-625-5772 | 电子邮件:victoria@adcap.ca
Forward-looking statements
前瞻性陈述
Certain statements made in this news release contain forward-looking information within the meaning of applicable securities laws, also known and/or referred to as "forward looking information" or "forward-looking statements". The words "anticipates", "believes", "expects", "will", "plans", "potential", and similar words, phrases, or expressions are often intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words, phrases, or expressions. Specific forward-looking statements contained in this news release include, but are not limited to, statements regarding the potential healthcare economic and other benefits of IXINITY and expectations regarding the product's prospects. These statements are based on factors or assumptions that were applied in drawing a conclusion or making a forecast or projection, including assumptions based on historical trends, current conditions, and expected future developments, and, in particular, Medexus's interpretation and assessment of the data presented by Medexus as summarized in this news release. Since forward-looking statements relate to future events and conditions, by their very nature they require making assumptions and involve inherent risks and uncertainties. Medexus cautions that, although the assumptions are believed to be reasonable in the circumstances, these risks and uncertainties mean that actual results could differ, and could differ materially, from the expectations contemplated by the forward-looking statements. Material risk factors include, but are not limited to, those set out in Medexus's materials filed with the Canadian securities regulatory authorities from time to time, including Medexus's most recent annual information form and management's discussion and analysis. In addition, specific risks and uncertainties relevant to the content of this news release include, among other things, the uncertainties inherent in research initiatives (including the possibility of unfavorable new data and further analyses of existing data); the risk that data are subject to differing interpretations and assessments by regulatory authorities and/or other relevant third parties; and whether regulatory authorities and/or other relevant third parties will be satisfied with the design and methodology of and results from the relevant study, which will depend on many factors, including determinations as to whether the product's benefits outweigh its known risks and determinations of the product's efficacy and cost-effectiveness in the context of a given facility (which varies by facility type). Accordingly, undue reliance should not be placed on these forward-looking statements, which are made only as of the date of this news release. Other than as specifically required by law, Medexus undertakes no obligation to update any forward-looking statements to reflect new information, subsequent or otherwise.
本新闻稿中的某些陈述包含适用证券法所指的前瞻性信息,也称为和/或称为 “前瞻性信息” 或 “前瞻性陈述”。“预期”、“相信”、“期望”、“将”、“计划”、“潜力” 等词语以及类似的词语、短语或表述通常用于识别前瞻性陈述,尽管并非所有前瞻性陈述都包含这些识别词、短语或表达方式。本新闻稿中包含的具体前瞻性陈述包括但不限于有关IXINITY潜在医疗经济和其他收益的陈述以及对该产品前景的预期。这些陈述基于在得出结论或进行预测或预测时适用的因素或假设,包括基于历史趋势、当前状况和预期未来发展的假设,尤其是本新闻稿中总结的Medexus对Medexus提供的数据的解释和评估。由于前瞻性陈述与未来事件和状况有关,因此就其本质而言,它们需要做出假设,并涉及固有的风险和不确定性。Medexus警告说,尽管人们认为这些假设在这种情况下是合理的,但这些风险和不确定性意味着实际结果可能与前瞻性陈述所设想的预期有所不同,甚至可能存在重大差异。重大风险因素包括但不限于Medexus不时向加拿大证券监管机构提交的材料中列出的因素,包括Medexus最新的年度信息表以及管理层的讨论和分析。此外,与本新闻稿内容相关的具体风险和不确定性包括研究计划固有的不确定性(包括可能出现不利的新数据和对现有数据的进一步分析);数据受到监管机构和/或其他相关第三方不同解释和评估的风险;以及监管机构和/或其他相关第三方是否会对相关研究的设计、方法和结果感到满意,将取决于许多因素,包括确定产品的收益是否超过其已知风险,以及确定该产品在给定设施的背景下(因设施类型而异)的功效和成本效益。因此,不应过分依赖这些前瞻性陈述,这些陈述仅在本新闻发布之日作出。除了法律的特别要求外,Medexus没有义务更新任何前瞻性陈述以反映后续或其他方面的新信息。
Additional notes
其他注意事项
IXINITY, as discussed in this news release, is a trademark of Medexus. Solely for convenience, trademarks and other protected names and marks in this news release may appear without the "", "", or similar symbols. Each such reference should be read as though it appears with the relevant symbol. Any such references are not intended to indicate, in any way, that the holder or holders of the relevant intellectual property rights will not assert those rights to the fullest extent under applicable law.
正如本新闻稿中所讨论的那样,IXINITY是Medexus的商标。仅为方便起见,本新闻稿中的商标和其他受保护的名称和标记可能不带有 “”、“” 或类似符号。每项此类参考文献都应看作是带有相关符号的文字。任何此类提法均无意以任何方式表明相关知识产权的持有人不会根据适用法律最大限度地主张这些权利。
The information in this news release is provided for informational purposes to investors in Medexus securities.
本新闻稿中的信息仅供Medexus证券投资者参考。
Medexus sponsored the study and publication discussed in this news release, and Medexus employees and paid consultants participated in the authoring and editing of the publication.
Medexus 赞助了本新闻稿中讨论的研究和出版物,Medexus 员工和带薪顾问参与了该出版物的创作和编辑。
Uniform resource locators, or website addresses, that appear in this news release are intended to be provided as inactive textual references only. Information contained on or accessible through these website addresses is not a part of this news release and is not incorporated by reference into this news release or any of Medexus's public filings.
本新闻稿中出现的统一资源定位符或网站地址仅作为非活跃文本参考提供。这些网站地址中包含或可通过这些网站地址访问的信息不是本新闻稿的一部分,也未以引用方式纳入本新闻稿或Medexus的任何公开文件中。