CSL Behring has added another major European market to the list of those providing reimbursement coverage of its haemophilia B gene therapy Hemgenix after agreeing an access deal in Germany.

CSL Behring 在与德国达成准入协议后，已将另一个主要欧洲市场加入到为其血友病 B 基因疗法 Hemgenix 提供报销覆盖的国家名单中。

The agreement with the GKV-Spitzenverband – the national association of statutory health insurance funds – means that

与法定健康保险基金的全国性协会GKV-Spitzenverband达成的协议意味着

Hemgenix

Hemgenix

(etranacogene dezaparvovec) can now be reimbursed across Germany's health system as a treatment for severe and moderately severe

(etranacogene dezaparvovec) 现已可以在德国的医疗系统中报销，作为治疗重度和中度重度疾病的药物。

haemophilia B

血友病B

in adult patients without a history of Factor IX (FIX) inhibitors.

在没有因子IX（FIX）抑制剂病史的成年患者中。

Hemgenix is the first gene therapy for this patient group to be approved in Europe and to date is also covered by the health systems of France, Denmark, Switzerland, Spain, the UK, and Austria, with the first patients already treated in France and Denmark.

Hemgenix是欧洲首个获批用于该患者群体的基因疗法，迄今为止，法国、丹麦、瑞士、西班牙、英国和奥地利的医疗系统也已覆盖该疗法，并且法国和丹麦已有首批患者接受治疗。

In Germany, the gene therapy is said to be the first covered under a national, success-based reimbursement model, with the cost of treatment – which is upwards of €3 million ($3.3 million) in EU countries – tied to its success in each individual patient.

在德国，这种基因疗法据说是在全国性的、基于成功的报销模式下首次被覆盖，治疗费用——在欧盟国家超过 300 万欧元（330 万美元）——与每位患者个体的成功效果挂钩。

'The performance-based payment model at [the] national level which was agreed with the GKV-Spitzenverband is unique in Germany,' said Stefan Neudoerfer, CSL Behring's chief negotiator in Germany. 'It addresses key reimbursement challenges, such as the question of long-term efficacy, which is inherent for any one-time therapy,' he added..

“与GKV-Spitzenverband达成的国家层面的基于绩效的支付模式在德国是独一无二的，”CSL Behring德国首席谈判代表斯特凡·诺伊多尔费尔表示。“它解决了关键的报销挑战，例如任何一次性疗法所固有的长期疗效问题，”他补充道。

Hemgenix is a one-shot infusion designed to correct the deficiency in FIX and remove or reduce the need for infusions with FIX replacement therapies every few days – which can cost hundreds of euros per year. Some patients treated with the therapy in trials have not needed to take FIX drugs for several years..

Hemgenix是一种一次性输液疗法，旨在纠正FIX的缺乏，并消除或减少每隔几天就需要进行FIX替代疗法的输液需求——这种疗法每年可能花费数百欧元。一些在试验中接受该疗法的患者已经多年无需服用FIX药物。

In February, new data from the HOPE-B trial – presented at the annual congress of the European Association for Haemophilia and Allied Disorders (EAHAD) – showed that after four years, 94% of patients remained FIX replacement-free with a 90% decrease in breakthrough bleeds to 0.4 per year.

今年 2 月，HOPE-B 试验的新数据在欧洲血友病及相关疾病协会 (EAHAD) 年会上发布，数据显示，经过四年时间，94% 的患者仍然无需 FIX 替代治疗，突破性出血事件减少了 90%，降至每年 0.4 次。

FIX activity in the body averaged over 37%, putting patients in the mild haemophilia range, with a level of 50% or more considered to be the normal range for people without haemophilia.

FIX 在体内的活性平均为 37%，使患者处于轻度血友病范围内，而 50% 或以上的水平被认为是无血友病人群的正常范围。

Hemgenix was approved by the European Commission in 2023 and has also been cleared in the US and several other world markets. CSL Behring, which launched the therapy in fiscal 2024, hasn't yet disclosed sales figures but said in February that uptake has 'accelerated' since it was introduced.

Hemgenix于2023年获得欧盟委员会批准，同时也在美国及其他多个国际市场获批。CSL Behring公司在2024财年推出了这一疗法，目前尚未披露销售数据，但其在二月表示，自产品推出以来，应用量“加速”增长。

The competitive picture for Hemgenix has improved since Pfizer abandoned its rival haemophilia B gene therapy

自从辉瑞放弃了其竞争对手的B型血友病基因疗法后，Hemgenix的竞争局面有所改善。

Beqvez

贝克维兹

(fidanacogene elaparvovec) in all world markets, saying weak demand made the business non-viable. It had been approved for marketing in the US, EU, and Canada last year.

(fidanacogene elaparvovec) 在全球所有市场，称疲软的需求使该业务无法维持。该药物去年已在美国、欧盟和加拿大获得上市批准。

Image by

图片由

Arek Socha

阿雷克·索查

from

从

Pixabay

Pixabay

.

。