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FDA批准新类Uptravi(selexipag)治疗肺动脉高压

2015-12-23 08:03:20 作者：新特药房来源：互联网浏览次数：0 文字大小：【大】【中】【小】

简介： 2015年12月21日，美国食品和药品监管局(FDA)批准Uptravi (selexipag)片治疗成年 with 肺动脉高压(PAH)，a慢性，逐步的，和使衰弱罕见的肺部疾病可导致死亡需要移植。FDA的药品评价和研究中心中药品评价I部 ...

关键字：selexipag商品名uptravi 治疗肺动脉高压孤儿药物指定

2015年12月21日，美国食品和药品监管局(FDA)批准Uptravi (selexipag)片治疗成年 with 肺动脉高压(PAH)，a慢性，逐步的，和使衰弱罕见的肺部疾病可导致死亡需要移植。
FDA的药品评价和研究中心中药品评价I部主任Ellis Unger，M.D.说：“Uptravi为有肺动脉高压患者提供一个另外治疗选择为，” “FDA支持为罕见病继续努力提供新治疗选择。”
PAH是在连接心脏至肺动脉发生高血压，它至心脏的右侧比正常更困难工作，可能导致远动能力受限制和气短，在其他更严重的并发症.
Uptravi属于一类药物称为口服IP前列环素受体激动剂。药物作用通过在血管壁放松肌肉扩张(开放)的血管和减低在血管中升高的压力供血至肺。
在一项1,156例有PAH参加者长期临床试验确定的安全性和疗效。与安慰剂比较，Uptravi被显示在减低为PAH住院有效和减低疾病进展的风险。在该试验参加者暴露于Uptravi中位时间1.4年。
在试验中用Uptravi患者观察到常见副作用包括头痛，腹泻，下颚痛，恶心，肌痛，呕吐，肢体痛，和脸红。
Uptravi被授予孤儿药物指定。孤儿药物指定提供激励例如税收减免，用户费用减免，和专有权资格帮助和鼓励对罕见病药物的开发。
Uptravi由总部在旧金山的美国Actelion Pharmaceuticals有限公司上市
Uptravi, selexipag (ACT-293987, NS-304)
Brand name: Uptravi
Generic name: selexipag
Company: Actelion Ltd.
Treatment for: Pulmonary Hypertension
Uptravi (selexipag) is a potent, orally available, selective IP prostacyclin receptor agonist indicated for the treatment of pulmonary arterial hypertension
New Drugs Online Report for selexipag
Information
Generic Name: selexipag
Trade Name: Uptravi
Synonym: ACT 293987
Entry Type: New molecular entity
Development and Regulatory status
UK: Pre-registration (Filed)
EU: Pre-registration (Filed)
US: Pre-registration (Filed)
UK launch Plans: Available only to registered users
Actual UK launch date:
Comments
Dec 14: Filed in the US [8].
04/03/2015 11:31:29
Dec 14: Actelion submits an application in the EU for selexipag as a treatment for PAH. The filing is based on data from the PIII GRIPHON study [7].
03/12/2014 11:06:29
Feb 10: First patient enrolled in PIII morbidity / mortality study at the end of 2009 [1].
15/02/2010 21:59:17
Trial or other data
Jun 14: Actelion announce that in a PIII study more than 1,000 patients, selexipag reduced risk of a morbidity/mortality event versus placebo by 39% [6].
18/06/2014 12:21:07
May 13: PIII study to continue after an independent interim analysis. Results expected mid-2014 [5].
09/05/2013 10:28:00
April 12: GRIPHON study due to complete Aug 13 [4]
20/04/2012 11:08:09
May 10: The PIII morbidity/mortality study is called GRIPHON, (Prostacyclin (PGI2) Receptor agonist in Pulmonary arterial Hypertension). It is a multicenter, double-blind, placebo-controlled study and is enrolling patients around the world. The primary endpoint of the trial the time to first clinical event of morbidity or mortality in patients with PAH. This trial is being conducted under an agreed SPA with the FDA [2].
23/05/2010 22:13:32
May 10: More data from the PII study in 43 patients were reported at the 2010 American Thoracic Society conference. Patients who received selexipag in addition to existing treatments had a 30.3% reduction in pulmonary vascular resistance (PVR) after 17 weeks (primary endpoint) vs placebo. Patients were initially given 200micrograms of selexipag twice a day, with the dose gradually raised in those who could tolerate it to 800mcg by day 21 of the trial. If side effects, such as jaw pain, pain in the extremities, or uncomfortable flushing occurred, the dose was reduced to the highest tolerated [2].
23/05/2010 22:13:03
Positive data have been obtained in a PIIa, placebo-controlled study to assess efficacy, safety and tolerability in 43 patients suffering from PAH and already receiving standard care with oral PAH medications. The primary endpoint of pulmonary vascular resistance change from baseline was met (p<0.01) [1].
15/02/2010 22:01:41
References
Available only to registered users
Category
BNF Category: Vasodilator antihypertensive drugs (02.05.01)
Pharmacology: non-prostanoid PGI2 receptor agonist
Epidemiology: In its pure idiopathic form it is a rare disease with an approximate annual incidence of about 1–3 cases per million population.2 There is a higher incidence of cases associated with connective tissue disease – about 10% of sufferers of CREST/scleroderma syndromes have the condition. [3]
Indication: Pulmonary hypertension
Method(s) of Administration
Oral
Company Information
Name: Actelion
US Name: Actelion
Further Information
Anticipated commissioning route (England) NHSE
High cost drug list? Yes
Tariff Not routinely commissioned by NHSE - IFR approval [9]
Implications Available only to registered users