EMA’s human medicines committee (CHMP) has recommended not renewing the conditional marketing authorisation for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy. Translarna is used in patients whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin gene and who are able to walk.The CHMP issued an initial negative opinion on the renewal of the marketing authorisation for Translarna in September 2023, which was confirmed in January 2024 following a re-examination requested by the company marketing the medicine.

欧洲药品管理局人类药物委员会（CHMP）建议不延长Translarna（ataluren）的有条件上市许可，Translarna是一种治疗杜兴氏肌营养不良症患者的药物。Translarna用于由肌营养不良蛋白基因中一种称为“无义突变”的遗传缺陷引起的疾病以及能够行走的患者。CHMP于2023年9月对Translarna上市许可证的续期发表了初步的负面意见，并于2024年1月在上市公司要求重新审查后得到确认。

Both rounds of assessment concluded that the effectiveness of Translarna had not been confirmed following a re-evaluation of the medicine’s benefits and risks.In May 2024, the European Commission asked the CHMP to further consider whether the data available on Translarna were sufficiently comprehensive to conclude on the medicine’s benefit-risk balance, and whether additional real-world data brought to the attention of the Commission during its decision-making process (including three recent publications) may impact the CHMP’s conclusion.

两轮评估都得出结论，在重新评估药物的益处和风险后，Translarna的有效性尚未得到证实。2024年5月，欧盟委员会要求CHMP进一步考虑Translarna上可用的数据是否足够全面，以得出该药物的利益-风险平衡的结论，以及在决策过程中提请委员会注意的其他现实数据（包括最近的三份出版物）是否会影响CHMP的结论。

In addition, following the appellate judgment of the Court of Justice of the European Union of 14 March 2024 in Case C-291/22 P, EMA decided to convene a new scientific advisory group on neurology (SAG) for Translarna. The assessment was therefore reset to this stage of the initial renewal procedure.In relation to the request from the European Commission, the CHMP reviewed the recent publications, one of which analysed the combined data of three clinical studies with Translarna already assessed by the CHMP (meta-analysis); a second evaluated the level of agreement amongst 12 clinicians on the use of Translarna;and a third described an initiative to .

此外，根据欧盟法院2024年3月14日对案件C-291/22 P的上诉判决，欧洲医学会决定为Translarna成立一个新的神经病学科学咨询小组（SAG）。因此，评估被重置为初始更新程序的这一阶段。关于欧盟委员会的要求，CHMP审查了最近的出版物，其中一篇分析了CHMP已经评估的三项临床研究与Translarna的综合数据（荟萃分析）；；第三个描述了一项倡议。

three recent publications: a meta-analysis of 3 clinical trials with Translarna (study 007, study 020 and study 041); an article on a newly-established registry bringing together data on rare neuromuscular disorders; and a study on the level of consensus amongst 12 clinicians on the use of Translarna;1,2,3additional information received from parents or caregivers, patient organisations, healthcare professionals organisations, and treating doctors;reports with subject-level data related to boys treated with Translarna.The CHMP carefully reviewed all this information and concluded that it did not bring sufficient evidence to confirm the effectiveness of the medicine.The Committee noted in particular that the meta-analysis, which was already reviewed and discussed by the CHMP, has several methodological shortcomings so its results cannot overrule the negative findings of the individual studies.

最近的三篇出版物：对3项Translarna临床试验的荟萃分析（研究007，研究020和研究041）；一篇关于新建立的登记册的文章，汇集了罕见神经肌肉疾病的数据；；1,2,3从父母或护理人员，患者组织，医疗保健专业人员组织和主治医生那里收到的其他信息；报告了与接受Translarna治疗的男孩有关的主题级数据。CHMP仔细审查了所有这些信息，并得出结论，它没有带来足够的证据来证实药物的有效性。委员会特别指出，CHMP已经审查和讨论过的荟萃分析在方法上存在一些缺陷，因此其结果不能推翻个别研究的负面结果。

The publication concerning rare neuromuscular disorders did not discuss the effectiveness of Translarna, while the publication regarding the level of agreement amongst neurologists did not provide new data on the medicine’s effectiveness.In addition, the CHMP took into account the views from a scientific advisory group on neurology, which comprised experts, including neurologists and people with lived experience of Duchenne muscular dystrophy.

关于罕见神经肌肉疾病的出版物没有讨论Translarna的有效性，而关于神经科医生之间协议水平的出版物没有提供有关该药物有效性的新数据。此外，CHMP还考虑了神经病学科学咨询小组的意见，该小组由专家组成，包括神经科医生和有杜兴氏肌营养不良症病史的人。

The group provided responses to specific questions posed by the CHMP.In reaching its opinion, the CHMP took all this information and the evidence accumulated on Translarna since its marketing authorisation into consideration.The Committee acknowledged the high unmet medical need for an effective treatment for patients with this rare disease; however, considering all the available evidence it concluded that the effectiveness of Translarna has n.

该小组对CHMP提出的具体问题做出了回应。CHMP在得出其意见时，考虑了Translarna自上市授权以来积累的所有这些信息和证据。委员会承认，对这种罕见疾病患者进行有效治疗的医疗需求尚未得到满足；然而，考虑到所有可用的证据，它得出结论，Translarna的有效性有n。

Related content

相关内容

EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine TranslarnaEMA confirms recommendation for non-renewal of authorisation of Duchenne muscular dystrophy medicine TranslarnaTranslarnaMeeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 June 2024.

EMA建议不续签Duchenne肌营养不良症药物TranslarnaEMA的授权，确认不续签Duchenne肌营养不良症药物TranslarnaTranslarnaMeeting的建议，来自人类使用药品委员会（CHMP）2024年6月24日至27日的亮点。

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外部内容

Judgment of the courtMeta Analysis Shows Slow Decline of Muscle Function With Ataluren for Nonsense Mutation DMDThe role of ataluren in the treatment of ambulatory and non-ambulatory children with nonsense mutation duchenne muscular dystrophyEURO-NMD registry: federated FAIR infrastructure, innovative technologies and concepts of a patient-centred registry for rare neuromuscular disorders.

courtMeta分析的判断显示，对于无意义突变DMD，Ataluren的肌肉功能缓慢下降。Ataluren在治疗无意义突变的门诊和非门诊儿童duchenne肌营养不良症中的作用欧洲NMD登记处：联邦公平基础设施，创新技术和以患者为中心的罕见神经肌肉疾病登记处的概念。

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