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CHMP建议批准Actelion肺动脉高压药物Opsumit

来源:生物谷 2013-10-28 08:18

2013年10月27日讯 /生物谷BIOON/ --Actelion公司10月25日宣布,实验性药物Opsumit(macitentan,10mg)获得了欧洲药品管理局(EMA)人用医药产品委员会(CHMP)的积极意见。CHMP建议批准Opsumit,作为单药疗法或联合其他药物,用于肺动脉高压(PAH)成人患者(WHO功能分级II-III)的长期治疗。

欧盟委员会(EC)将于2个月内做出审查决定。

CHMP的积极意见,是基于里程碑意义的III期 SERAPHIN研究的疗效和安全性数据。该项研究表明,与安慰剂相比,Opsumit(macitentan,10mg)治疗组发病率/死亡率事件风险降低45%(p<0.0001),达到了研究的主要终点。所观察到的疗效,与患者是否已接受其他PAH疗法无关。此外,与安慰剂相比,Opsumit治疗组PAH相关住院或死亡风险降低50%(p<0.0001)。

SERAPHIN研究中,Opsumit治疗组所观察到的最常见不良反应为:贫血、鼻咽炎/咽炎、支气管炎、头痛、感冒、尿路感染。

FDA于今年10月18日批准Opsumit(macitentan,10mg),作为每日一次的疗法,用于治疗肺动脉高压(PAH),以延缓疾病进展。Opsumit在其他国家的监管审查正在进行中。

Opsumit属于一类名为内皮素受体拮抗剂的药物,能够放松肺动脉并降低血压,Opsumit与该类药物中的其他药物一样,具有一个黑框警示,指出该药不可用于孕妇,因为Opsumit可能对胎儿造成伤害。

Opsumit是Actelion公司PAH药物Tracleer的继任者,Tracleer将于2015年失去专利保护。根据Actelion提供的数据,Tracleer在2012年的销售达15亿瑞士法郎,为该公司最畅销的药物。Actelion正指望Opsumit来弥补Tracleer专利到期所致的销售预期下降。

Opsumit将与市面上的其他PAH药物展开竞争,包括吉利德(Gilead)的Letairis,该药在美国以外国家和地区由葛兰素史克(GSK)以品牌名Volibris销售。

肺动脉高压(PAH)是一种极度严重的疾病,症状包括:呼吸短促、易于疲劳、晕厥、胸痛以及腿部和踝部水肿。此外,患者的肺动脉高压会逐步加重,甚至使寿命缩短。多数肺动脉高压相关的症状源自右心衰竭。(生物谷Bioon.com)

英文原文:ACTELION RECEIVES POSITIVE CHMP OPINION FOR OPSUMIT (MACITENTAN) FOR THE LONG-TERM TREATMENT OF PULMONARY ARTERIAL HYPERTENSION

-CHMP adopts positive opinion for Opsumit? (macitentan), as monotherapy or in combination, for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class II to III

-European Commission decision granting marketing authorization in the EU expected in two months

ALLSCHWIL, SWITZERLAND - 25 October 2013 - Actelion Ltd (SIX: ATLN) announced today that the Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMA), issued a positive opinion for the use of Opsumit? (macitentan) 10mg for the treatment of pulmonary arterial hypertension (PAH).

The CHMP recommended that the European Commission approves Opsumit?, as monotherapy or in combination, for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class II to III. Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease.

A CHMP positive opinion is one of the final steps before marketing authorization is granted by the European Commission. The European Commission is expected to issue a final decision in two months.

Dr Nazzareno Galiè from the Institute of Cardiology, University of Bologna, Bologna, Italy commented "With Opsumit? we have the first demonstration of delayed disease progression in PAH patients over long-term. Opsumit? was tested in the first ever outcome driven clinical trial from which we now have evidence that Opsumit? is effective in both treatment naive patients as well as those already taking PDE5-inhibitors. The opinion of the CHMP today is a very encouraging step for patients with PAH."

The CHMP's positive opinion was based on the review of efficacy and safety data from the landmark phase III SERAPHIN study. Published in the New England Journal of Medicine in August 2013, the SERAPHIN study showed the risk of a morbidity/mortality event - the primary endpoint of the study - was reduced by 45% (p<0.0001) with macitentan 10mg compared to placebo. This effect was observed irrespective of whether or not patients were already treated with other therapies for PAH. SERAPHIN also showed a reduction in risk of PAH related hospitalization or death of 50% (p<0.0001) compared to placebo.

Gérald Simonneau M.D., Professor of Pneumology and Head of the Department of Pneumology and Intensive Care Unit, H?pital Kremlin Bicêtre, Paris-Sud University, France, commented; "In 2008 we, the experts in the field of PAH, proposed that clinical trials used morbidity and mortality as a primary endpoint in future PAH studies. Today, Opsumit?, is the first drug that was tested with this endpoint and it has now received a positive opinion from CHMP. I consider this as a turning point for PAH and believe that this robust and clinically relevant standard should be applied in all future clinical trials."

The most common adverse reactions observed in patients treated with Opsumit? were anemia, nasopharyngitis / pharyngitis, bronchitis, headache, influenza, and urinary tract infection.

Opsumit? is approved in the US, and has been submitted to regulatory bodies worldwide for the treatment of patients with PAH. Regulatory reviews by health authorities in other countries are ongoing.

PAH is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented, "We are delighted by the positive opinion issued by the CHMP on Opsumit?. If approved, Opsumit? could transform the lives of many people living with PAH. This positive opinion stands testament to the efforts of our development teams and our ongoing commitment to patients living with PAH."

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