CAMBRIDGE, England--(BUSINESS WIRE)--Napp Pharmaceuticals Limited, a member of the international network of Mundipharma independent associated companies, today announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has authorised rezafungin for the treatment of invasive candidiasis in adults in Great Britain (GB).1,2.

英国剑桥——（商业新闻短讯）——Mundipharma独立关联公司国际网络成员Napp Pharmaceuticals Limited今天宣布，药品和保健品管理局（MHRA）已授权雷扎芬净治疗英国（GB）成人侵袭性念珠菌病[1,2]。

The authorisation was based on positive results from the pivotal ReSTORE Phase III clinical trial which demonstrated statistical non-inferiority for rezafungin, dosed once weekly, when compared to the current standard of care, caspofungin, dosed once daily.2,3,4 These findings are supported by the STRIVE Phase II clinical trial and an extensive nonclinical development program.3,4.

该授权基于关键恢复III期临床试验的积极结果，该试验表明，与目前的护理标准卡泊芬净相比，每周服用一次的雷扎芬净具有统计学上的非劣效性，每天服用一次。2,3,4这些发现得到了STRIVE II期临床试验和广泛的非临床开发计划的支持。

ReSTORE, a multicentre, prospective, randomised and double-blind Phase III trial compared the efficacy and safety of intravenous rezafungin with intravenous caspofungin in eligible patients with invasive candidiasis.2,3 The primary efficacy outcome was global response (confirmed by the Data Review Committee [DRC]) at day 14.3 Global response was determined from clinical response, mycological response, and radiologic response (for qualifying subjects with invasive candidiasis).2,3 Of the 187 patients in the modified intention-to-treat (mITT) population, 59.1% of patients in the rezafungin group (55/93) and 60.6% of patients in the caspofungin group (57/94) had a global response of cure at day 14 (weighted treatment difference −1.1% [95% CI −14.9, 12.7].2,3 Non-inferiority was to be concluded if the lower bound of the 95% confidence internal (CI) for the difference in Day 14 cure rates (rezafungin – caspofungin) was >-20%.2.

ReSTORE是一项多中心，前瞻性，随机和双盲的III期临床试验，比较了静脉注射瑞扎芬净和静脉注射卡泊芬净对符合条件的侵袭性念珠菌病患者的疗效和安全性。2,3主要疗效结果是第14.3天的全球反应（由数据审查委员会（DRC）证实），全球反应由临床反应确定，真菌学反应和放射学反应（适用于符合条件的侵袭性念珠菌病患者）。2,3在改良意向治疗（mITT）人群中的187名患者中，雷扎芬净组59.1%（55/93）和卡泊芬净组60.6%（57/94）的患者在第14天有整体治愈反应（加权治疗差异-1.1%[95%CI-14.9，12.7]。2,3如果第14天治愈率差异（雷扎芬净-卡泊芬净）的95%置信区间（CI）的下限>20%，则可以得出非劣效性结论。

Rezafungin was generally well tolerated in clinical trials, to date very common adverse reactions include hypokalaemia, pyrexia, and diarrhoea. Common adverse reactions include anaemia, hypomagnesaemia, hypophosphataemia, hypotension, wheezing, vomiting, nausea, abdominal pain, constipation, erythema, and rash.

雷扎芬净在临床试验中通常耐受性良好，迄今为止，非常常见的不良反应包括低钾血症，发热和腹泻。常见的不良反应包括贫血、低镁血症、低磷血症、低血压、喘息、呕吐、恶心、腹痛、便秘、红斑和皮疹。

Increased blood alkaline phosphatase, hepatic enzymes, alanine aminotransferase, aspartate aminotransferase and blood bilirubin were also common. Uncommon adverse events were hyperphosphataemia, hyponatraemia, phototoxicity, tremor and increased eosinophil count. There were spontaneous reports of urticaria (frequency not known).

血液碱性磷酸酶，肝酶，丙氨酸转氨酶，天冬氨酸转氨酶和血液胆红素升高也很常见。不常见的不良事件是高磷血症，低钠血症，光毒性，震颤和嗜酸性粒细胞计数增加。有自发性荨麻疹的报道（频率未知）。

Transient infusion-related reactions have occurred with rezafungin, characterised by flushing, sensation of warmth, nausea, and chest tightness.2.

雷扎芬净发生了短暂的输液相关反应，其特征是潮红，温暖感，恶心和胸闷。

Rezafungin may cause increased risk of phototoxicity. Eligible patients should be advised to avoid sun exposure and other sources of UV radiation without adequate protection during treatment and for 7 days after the last administration of rezafungin.2

Rezafungin可能导致光毒性风险增加。应建议符合条件的患者在治疗期间和最后一次服用热扎芬净后7天内避免阳光照射和其他紫外线辐射源，而没有足够的保护。2

Invasive candidiasis, is a severe, life-threatening infection of the bloodstream and/or deep/visceral tissues.10,11 It affects seriously ill people, especially those with a weakened immune system and the mortality rate can be 40% or more.12,13 It can place a large burden on the healthcare system, with the potential for extended treatment regimens and long hospital stays.14 In the last 15 years there have been no new treatments and morbidity and mortality rates remain largely the same, indicating the need for alternative treatment options.5-9,15.

侵袭性念珠菌病是一种严重的危及生命的血流和/或深部/内脏组织感染。10,11它影响重症患者，尤其是免疫系统受损的患者，死亡率可能达到40%或更高。12,13它可能给医疗保健系统带来沉重负担，有可能延长治疗方案和住院时间.14在过去的15年中，没有新的治疗方法，发病率和死亡率基本保持不变，表明需要替代治疗方案[5-9,15]。

Professor P. Lewis White PhD, FECMM, FRCPath, Consultant Clinical Scientist and Head of the PHW Mycology Reference Laboratory, Honorary Professor, Cardiff University Centre for Trials Research said “Invasive candidiasis remains a difficult-to-treat disease which has a significant impact on critically ill and immunocompromised patients.

卡迪夫大学试验研究中心名誉教授、临床顾问科学家兼PHW真菌学参考实验室主任、FECMM、FRCPath教授P.Lewis White博士表示，“侵袭性念珠菌病仍然是一种难以治疗的疾病，对重症患者和免疫功能低下患者产生重大影响。

With limited advances in treating this condition over the past 15 years, today’s announcement is a positive step towards supporting eligible patients and could enable the healthcare professional community an alternative way to manage these patients with invasive candidiasis using a new, once-weekly intravenous treatment option.”.

在过去的15年中，治疗这种疾病的进展有限，今天的宣布是支持符合条件的患者的积极一步，并且可以使医疗保健专业社区成为使用新的，每周一次的静脉注射治疗选择来管理这些侵袭性念珠菌病患者的替代方法。”。

“This authorisation is a culmination of years of developing an additional treatment option for eligible adult patients suffering from invasive candidiasis and underscores our commitment to supporting management of infectious diseases,” said Dr Ben David, Medical Director for Northern Europe at Mundipharma..

Mundipharma北欧医疗主管Ben David博士说：“这项授权是多年来为患有侵袭性念珠菌病的符合条件的成年患者开发额外治疗选择的高潮，并强调了我们支持传染病管理的承诺。”。。

Rezafungin has been granted Orphan Drug Designation (ODD) in GB for the treatment of invasive candidiasis in adults.1 The MHRA grants ODD to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 5 in 10,000 people in GB where no satisfactory method of diagnosis, prevention or treatment of the condition concerned exists, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.16.

Rezafungin已被授予GB孤儿药名称（ODD），用于治疗成人侵袭性念珠菌病。1 MHRA授予ODD用于治疗、诊断或预防罕见疾病或疾病的药物，这些疾病或疾病在GB中影响不到10000人中的5人，其中不存在令人满意的诊断、预防或治疗相关疾病的方法，或者，如果存在这种方法，这种药物必须对那些受疾病影响的人有显着的益处。

*ENDS*

*结束*

About invasive candidiasis

关于侵袭性念珠菌病

Invasive candidiasis (IC) continues to be an area of significant unmet need, especially for critically ill patients in hospitals and patients with compromised immune systems.14 Despite a number of available treatments, the mortality rate for patients with invasive candidiasis can be 40% or more.13,14 IC is characterised as a severe, life-threatening systemic Candida infection of the bloodstream and/or deep/visceral tissues.17.

侵袭性念珠菌病（IC）仍然是一个尚未满足需求的领域，特别是对于医院的重症患者和免疫系统受损的患者。14尽管有许多可用的治疗方法，但侵袭性念珠菌病患者的死亡率可能为40%或更多。13,14 IC的特征是严重，危及生命的血液和/或深部/内脏组织的系统性念珠菌感染。

About Rezafungin

关于Rezafungin

Rezafungin is a novel once-weekly echinocandin that has been developed for the treatment of invasive candidiasis in adults.18

Rezafungin是一种每周一次的新型棘白菌素，已被开发用于治疗成人侵袭性念珠菌病

About Napp

关于Napp

Napp Pharmaceuticals Limited is part of Mundipharma, a global healthcare company with a presence across Africa, Asia Pacific, Canada, Europe, Latin America, and the Middle East.

Napp Pharmaceuticals Limited是Mundipharma的一部分，Mundipharma是一家全球性医疗保健公司，业务遍及非洲、亚太地区、加拿大、欧洲、拉丁美洲和中东。

Mundipharma is dedicated to bringing innovative treatments to patients in the areas of pain management, infectious disease and consumer healthcare as well as other severe and debilitating disease areas. Our guiding principles, centered around Integrity and Patient-Centricity, are at the heart of everything we do.

Mundipharma致力于在疼痛管理、传染病和消费者保健以及其他严重和衰弱疾病领域为患者提供创新治疗。我们的指导原则以正直和以病人为中心为中心，是我们所做一切的核心。

For more information visit www.mundipharma.com.

有关更多信息，请访问www.mundipharma.com。

Future clinical trial data

未来的临床试验数据

A second Phase III clinical trial of rezafungin for the prevention of invasive fungal disease in patients undergoing allogeneic blood and marrow transplantation (ReSPECT trial) is currently being conducted.18

目前正在进行rezafungin预防异基因血液和骨髓移植患者侵袭性真菌病的第二阶段III期临床试验（REPECT试验）

Registered office addresses

注册办公地址

NAPP PHARMACEUTICALS LIMITED a company having its registered office at Cambridge Science Park, Milton Road, Cambridge CB4 0AB, England.

NAPP PHARMACEUTICALS LIMITED，一家注册办事处位于英国剑桥CB4 0AB弥尔顿路剑桥科技园的公司。

MUNDIPHARMA RESEARCH LIMITED, a company having its registered office at Cambridge Science Park, Milton Road, Cambridge, CB4 0AB, England.

MUNDIPHARMA RESEARCH LIMITED，一家注册办事处位于英国剑桥CB4 0AB弥尔顿路剑桥科技园的公司。

® REZZAYO is a Registered Trademark of Cidara Therapeutics, Inc, used under license.

®REZZAYO是Cidara Therapeutics，Inc的注册商标，根据许可证使用。

References

参考文献

1. UK Medicines and Healthcare Products Regulatory Agency authorisation. Available at: https://www.gov.uk/government/news/rezzayo-approved-to-treat-adult-patients-with-invasive-candidiasis (last accessed February 2024)

1、英国药品和保健品管理局授权。网址：https://www.gov.uk/government/news/rezzayo-approved-to-treat-adult-patients-with-invasive-candidiasis（上次访问时间为2024年2月）

2. REZZAYO® Summary of Product Characteristics.

2、REZZAYO®产品特性总结。

3. Thompson GR, et al. Lancet 2023;401(10370):49–59.

3.Thompson GR等人，《柳叶刀》2023；401（10370）：49-59。

4. Thompson GR, et al. Lancet Infect Dis 2023; https://doi.org/10.1016/S1473-3099(23)00551-0

Thompson GR等人，《柳叶刀感染DIS 2023》；https://doi.org/10.1016/S1473-3099（23）00551-0

5. Ham YY, et al. Future Microbiology 2021;16(1):27–36.

5.Ham YY等人，《未来微生物学2021》；16（1）：27-36。

6. CANCIDAS (formerly Caspofungin MSD) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/cancidas-previously-caspofungin-msd (last accessed February 2024).

CANCIDAS（前Caspofungin MSD）EPAR。网址：https://www.ema.europa.eu/en/medicines/human/EPAR/cancidas-previously-caspofungin-msd（上次访问时间为2024年2月）。

7. Vfend (voriconazole) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/vfend (last accessed February 2024).

7.Vfend（伏立康唑）EPAR。网址：https://www.ema.europa.eu/en/medicines/human/EPAR/vfend（上次访问时间为2024年2月）。

8. Ecalta (anidulafungin) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/ecalta (last accessed February 2024).

8.Ecalta（anidulafungin）EPAR。网址：https://www.ema.europa.eu/en/medicines/human/EPAR/ecalta（上次访问时间为2024年2月）。

9. Mycamine (micafungin) EPAR. Available at: https://www.ema.europa.eu/en/medicines/human/EPAR/mycamine (last accessed February 2024).

9.Mycamine（米卡芬金）EPAR。网址：https://www.ema.europa.eu/en/medicines/human/EPAR/mycamine（上次访问时间为2024年2月）。

10. Pappas PG, et al. Nat Rev Dis Primers 2018;4:18026.

10.Pappas PG等人，Nat Rev Dis Primers 2018；4:18026.

11. CDC. Invasive candidiasis. Available at: https://www.cdc.gov/fungal/diseases/candidiasis/invasive/definition.html (last accessed February 2024).

疾病预防控制中心。侵袭性念珠菌病。网址：https://www.cdc.gov/fungal/diseases/candidiasis/invasive/definition.html（上次访问时间为2024年2月）。

12. Kullberg BJ, Arendrup MC. N Engl J Med 2015;373:1445–56.

Collberg BJ，Arendrup MC.N Engl J Med 2015；373:1445-56.

13. Bassetti M, et al. Crit Care 2019;23(1):219.

13.Bassetti M等人，《Crit Care 2019》；23（1）：219。

14. Cortegiani A, et al. Cochrane Database Syst Rev 2016;2016:Cd004920.

14.Cortegiani A等人，Cochrane数据库系统2016年修订版；2016年：Cd004920。

15. Bassetti M, et al. J Antimicrob Chemother 2018; 73 Suppl 1: i14–i25 doi:10.1093/jac/dkx445.

15.Bassetti M等人J Antimicrob Chemother 2018；73增刊1:i14-i25 doi:10.1093/jac/dkx445。

16. UK Medicines and Healthcare Products Regulatory Agency, Guidance – Orphan medicinal products. Available at: https://www.gov.uk/guidance/orphan-medicinal-products-in-great-britain (last accessed February 2024).

16、英国药品和保健品管理局，指南-孤儿药品。网址：https://www.gov.uk/guidance/orphan-medicinal-products-in-great-britain（上次访问时间为2024年2月）。

17. Cortes JA, Corrales IF. Invasive candidiasis: Epidemiology and Risk Factors. November 2018. Available at https://www.intechopen.com/chapters/64365 (last accessed February 2024).

17.Cortes JA，Corrales IF。侵袭性念珠菌病：流行病学和危险因素。2018年11月。可在https://www.intechopen.com/chapters/64365（上次访问时间为2024年2月）。

18. Cidara Therapeutics and Mundipharma Announce Positive Topline Results from the Global Phase 3 Pivotal ReSTORE Trial of Rezafungin for the Treatment of invasive candidiasis. Available at https://www.mundipharma.com/Cidara-Therapeutics-and-Mundipharma-Announce-Positive-Topline-Results-from-the-Global-Phase-3-Pivotal-ReSTORE-Trial-of-Rezafungin-for-the-Treatment-of-Candidemia-and-Invasive-Candidiasis (last accessed February 2024)..

18.Cidara Therapeutics和Mundipharma宣布Rezafungin治疗侵袭性念珠菌病的全球3期关键恢复试验的阳性结果。可在https://www.mundipharma.com/Cidara-Therapeutics-and-Mundipharma-Announce-Positive-Topline-Results-from-the-Global-Phase-3-Pivotal-ReSTORE-Trial-of-Rezafungin-for-the-Treatment-of-Candidemia-and-Invasive-Candidiasis（上次访问时间为2024年2月）。。

Job code: UK-RZF-2300053

工作代码：UK-RZF-2300053

Date of preparation: February 2024

编制日期：2024年2月