| 2015年9月11日，欧盟委员会批准Santhera的首款药物Raxone，用于Leber遗传性视神经病变（LHON）青少年及成年患者的视觉障碍治疗。这种罕见病症是一种遗传性的线粒体疾病，如果不进行治疗，它通常会使健康患者快速、深刻及永久性失明。 数据证明，Raxone治疗可以阻止患者进一步的视力丧失，能够促进临床相关的视力恢复，Santhera首席执行官 Meier 博士称。对Santhera来说，此次Raxone作为一款有效的治疗药物在欧洲获批用于LHON及作为首款获批用于线粒体疾病是一个重要的里程碑，因为它标志着我们转变成一个有产品上市的制药公司。 Raxone是线粒体疾病治疗领域的一项重大突破 Raxone在线粒体疾病治疗领域代表了一项重大突破，它的批准为 LHON 患者获得治疗、实现有意义的视力改善铺平了道路，德国慕尼黑大学神经学教授 Klopstock称，他也是线粒体疾病德国网络的协调者及一名LHON研究者。LHON是一种特别灾难性的疾病，因为遭受这种疾病的患者不健康，并通常比较年轻，他们在几个月内会迅速双眼失明。如果不进行治疗，多数患者仍会持续失明。 Raxone是一种口服药物，每天的用药剂量为900mg（每天三次，每次2片，随食物一起服用），用于青少年及成年LHON患者视觉障碍治疗。疗效数据基于 Santhera 的随机、安慰剂对照 RHODOS试验及开放式扩大获取项目，这两项数据一起证明在Raxone治疗患者中视力丧失是可以缓解或逆转的。 New Drugs Online Report for idebenone Information Generic Name: idebenone Trade Name: Raxone Synonym: Catena; Raxone; SNT MC17; SNT-MC17; SNTMC17; Sovrima Entry Type: Licence extension / variation Development and Regulatory status UK: Phase III Clinical Trials EU: Phase III Clinical Trials US: Phase III Clinical Trials UK launch Plans: Available only to registered users Actual UK launch date: Comments Apr 15: FDA has granted idebenone fast track status for DMD [9]. 12/01/2016 10:44:09 Mar 15: discussion with regulatory agencies continuing. [7] 11/03/2015 18:04:27 Apr 14: Santhera Pharmaceuticals reported in September 2013 that it was continuing to explore strategic options and to seek additional financing [5] 15/04/2014 16:16:48 Orphan drug status in EU and US [2]. 05/09/2009 19:19:47 Both the FDA and EMEA have agreed that, subject to positive outcome, a single pivotal study could suffice for approval [1]. 05/09/2009 19:16:49 Sep 09: PIII study started [1]. 05/09/2009 19:16:15 Trial or other data Current options (corticosteroids) can delay but not prevent loss of walking ability. If licensed, idebenone would potentially be suitable for all affected patients with DMD. Likely to be expensive. 01/10/2015 15:06:13 Apr 15. The PIII DELOS study published in the Lancet. In this 1-year study of 64 patients (10-18 years) with Duchenne muscular dystrophy treatment with idebenone (300mg three times a day) attenuated the fall in peak expiratory flow (as percentage predicted) versus placebo (difference of -6.27%; [0.61 to 11.93]; p=0.031). The effect of idebenone on respiratory function outcomes was similar between patients with previous corticosteroid use and steroid-naive patients. [8] 22/04/2015 16:49:07 May 14: Santhera Pharmaceuticals (SIX:SANN) announces today that its PIII DELOS study met the primary endpoint of delaying the loss of respiratory function compared to placebo; difference between Catena®/Raxone® and placebo in the change from baseline to week 52 in Peak Expiratory Flow (p=0.04). [6] 15/05/2014 12:07:54 Apr 13: The PIII DELOS study has passed a planned futility and safety analysis; the Data Safety Monitoring Board considers the study has a reasonable chance of achieving its primary endpoint for improving or delaying the loss of respiratory function in Duchenne patients not using corticosteroids [4]. 17/04/2013 16:27:13 May 11: Following a 12-month PII DELPHI study, participants were able to enter the DELPHI-E, a 24-month open-label study, and 19 did so. They received weight-adjusted doses of Catena® at 450/900 mg/day. On average, patients did not receive Catena® for 21 months between DELPHI and DELPHI-E studies. During this period, PEF, FVC and maximum inspiratory mouth pressures (MIP) declined. The rate of decline was numerically lower for PEF and MIP when patients received Catena® treatment during the DELPHI-E study vs the period where they did not receive Catena®. There was no change in the rate of decline in FVC between the off-medication period and the DELPHI-E study period. Comparing data from DELPHI-E with natural history data for Duchenne Muscular Dystrophy, patients in the DELPHI-E study performed better in PEF for ages 16 and older and in FVC for ages 18 and older. Catena® possibly slows the progression of respiratory dysfunction in these age periods [3]. 12/05/2011 13:08:24 The PIIa DELPHI trial (SNT-II-001; NCT00654784) in 21 boys aged 8-16 years has demonstrated positive effects on cardiac and respiratory parameters with no difference in safety and tolerability vs idebenone and placebo. The trial is expected to complete in Feb 2011 [2]. 05/09/2009 19:25:31 Takeda have an exclusive right to commercialise idebenone in the EU and Switzerland under the brand name Sovrima [1]. 05/09/2009 19:17:52 Sep 09: The 12-month PIII DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) study has started and will enroll up to 240 patients (ambulatory and non-ambulatory) aged 10 to 18 years in 25 centres in EU and US and will compare idebenone 900 mg/day vs placebo. The primary endpoint is the change from baseline to week 52 in respiratory function measured by peak expiratory flow. Secondary endpoints include other respiratory parameters, muscle strength and motor function over the treatment period and improvement in quality of life . 05/09/2009 19:17:31 References Available only to registered users Category BNF Category: Drugs used in neuromuscular disorders (10.02) Pharmacology: Mitochondrial membrane transport protein stimulant Epidemiology: One of the most common and devastating types of muscular degeneration. Affects boys of all ethnicities from as early as 3-5 years of age with av life expectancy of 30-35 years. About 30,000 males in Europe and N. America have DMD [1] Indication: Duchenne muscular dystrophy Additional Details: first-line Method(s) of Administration Oral Company Information Name: Santhera US Name: Santhera Further Information Anticipated commissioning route (England) NHSE High cost drug list? Yes Implications Available only to registered users |
欧盟批准Raxone用于治疗Leber遗传性视神经病变简介：
2015年9月11日，欧盟委员会批准Santhera的首款药物Raxone，用于Leber遗传性视神经病变（LHON）青少年及成年患者的视觉障碍治疗。这种罕见病症是一种遗传性的线粒体疾病，如果不进行治疗，它通常会使健康患 ... 责任编辑：admin
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