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首个干细胞治疗药Holoclar获CHMP建议批准积极意见

来源:生物谷 2014-12-23 10:12

2014年12月22日讯/生物谷BIOON/--近期,欧洲药品管理局人用药产品委员会(Committee for Medicinal Products for Human Use,CHMP)向欧盟委员会(EC)推荐批准7个新药,而其中最引人注目的是首个干细胞治疗药物。

意大利凯西制药公司(Chiesi Farmaceutici)的孤儿药Holoclar 是获批的首个包含干细胞的先进治疗产品,用于治疗成人因物理或化学因素造成的中至重度的角膜缘干细胞缺损。

Holoclar也是首个用于治疗角膜缘干细胞缺损的药物,角膜缘干细胞缺损是一种罕见的眼科疾病,可以导致失明,Holoclar不仅可以成为角膜移植的替代疗法,也可以在大范围眼部损伤的情况下增加角膜移植成功的概率。

除了干细胞治疗Holoclar之外,此次获得CHMP建议批准积极意见的还有Orexigen Therapeutic的 Mysimba(纳曲酮/丁氨苯丙酮)。该药物用于肥胖症患者的体重管理,以及患有肥胖相关并发症的患者。此前的临床试验已经证明服用Mysimba的肥胖症患者与服用安慰剂的对照组相比体重确实下降了,这是此番获得推荐重要依据。

在Mysimba的安全性方面,CHMP表示尽管目前还没有确凿的数据可以证明长期服药是否会对心脏造成影响,然而以目前的临床数据显示,Mysimba是可以安心服用的,并且在不远的将来,进一步评估药物安全性的试验会展开,用来确认该药物对心血管的安全性。尽管如此,CHMP还是建议患者在服药16周后接受体检,只有当体重至少下降5%时,才能够继续服药。

除了上述两种药品之外,Zambon用于治疗帕金森症的Xadago(沙芬酰胺);Durata Therapeutics用于治疗皮肤感染的Xydalba(达巴万星);Aptalis Pharma用于治疗囊性纤维化伴慢性铜绿假单胞菌感染的孤儿药 Quinsair (左氧氟沙星);用于预防心肌梗塞、缺血性中风、外周动脉病变、急性冠脉综合征、房颤栓塞的仿制药Clopidogrel ratiopharm(氯吡格雷)均获得CHMP建议批准的积极意见。

最后, Genzyme Europe 用于控制成人透析后发生高磷血症的Tasermity (盐酸司维拉姆)也获得了知情同意程序。知情同意程序是指授权药获得用药参考权,并且该参考药的市场营销授权持有者被授予在病人用药程序中为其提供相关建议的权利。

CHMP今年已经向欧盟委员会推荐批准了82种新药。(生物谷Bioon.com)

原文First stem-cell therapy among seven drugs up for EU nod

The first stem-cell therapy has been recommended for approval by the Committee for Medicinal Products for Human Use, among seven new medicines to clear one of the last hurdles to market.

Chiesi Farmaceutici’s orphan drug Holoclar has been given the thumbs up - as the first advanced therapy medicinal product (ATMP) containing stem cells - for the treatment of moderate-to-severe limbal stem cell deficiency due to physical or chemical burns to the eyes in adults.

It is also the first medicine recommended for LSCD - a rare eye condition that can result in blindness - potentially offering an alternative to transplantation and increasing the chances of a successful corneal transplant where the injury has caused extensive eye damage, the CHMP said.

A positive opinion was also issued for Orexigen Therapeutic’s Mysimba (naltrexone/bupropion) for weight management in adults who are obese, or those overweight with one or more related complications, alongside a reduced-calorie diet and physical activity.

The endorsement comes on the back of clinical trials showing that more patients treated with Mysimba achieved clinically-relevant weight loss than patients treated with placebo.

On the safety side, the Committee noted that uncertainties remain over the drug’s long-term affect on the heart, but stressed that interim clinical data was “reassuring” and that a second study is planned in order to continue monitoring cardiovascular safety. Nevertheless, it has recommended that patients should be monitored after 16 weeks’ treatment and only allowed to continue treatment if they have achieved at least 5% weight loss.

Elsewhere, recommendations were handed out for: Zambon’s Xadago (safinamide) for Parkinson’s disease; Durata Therapeutics’ Xydalba (dalbavancin) for the treatment of skin and skin structure infections; Aptalis Pharma’s orphan drug Quinsair (levofloxacin) for the treatment of chronic pulmonary infections due to Pseudomonas aeruginosa in adult patients with cystic fibrosis; and generic medicine Clopidogrel ratiopharm (clopidogrel) for prevention of myocardial infarction, ischaemic stroke, peripheral arterial disease and acute coronary syndrome, as well as atherothrombotic and thromboembolic events in atrial fibrillation.

Finally, a positive opinion was issued for Genzyme Europe’s informed consent application for Tasermity (sevelamer hydrochloride) for the control of hyperphosphataemia in adults receiving dialysis. In an informed consent application, reference is made to an authorised medicine and the marketing authorisation holder of the reference medicine has given consent to the use of their dossier in the application procedure.

The CHMP has now recommended 82 new medicines for approval in Europe this year.

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