Actelion公司于2013年12月20日宣布，药物Opsumit（macitentan，10mg）已获欧盟委员会（EC）批准，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。
Opsumit的获批，部分基于具有里程碑意义的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。
此前，Opsumit已分别于今年10月和11月获FDA和加拿大卫生部批准，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。
Opsumit(macitentan) 10mg
1. Name of the medicinal product
Opsumit 10 mg film-coated tablets.
2. Qualitative and quantitative composition
Each film-coated tablet contains 10 mg macitentan.
Excipients with known effect:
Each film-coated tablet contains approximately 37 mg of lactose (as monohydrate) and approximately 0.06 mg of lecithin (soya) (E322).
For the full list of excipients, see section 6.1.
3. Pharmaceutical form
Film-coated tablet (tablet).
5.5 mm, round, biconvex, white to off-white film-coated tablets, debossed with “10” on one side.
4. Clinical particulars
4.1 Therapeutic indications
Opsumit, as monotherapy or in combination, is indicated for the long-term treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) 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 (see section 5.1).
4.2 Posology and method of administration
Treatment should only be initiated and monitored by a physician experienced in the treatment of PAH.
Posology
Opsumit is to be taken orally at a dose of 10 mg once daily, with or without food. The film-coated tablets are not breakable and are to be swallowed whole, with water.
Opsumit should be taken every day at about the same time. If the patient misses a dose of Opsumit, the patient should be told to take it as soon as possible and then take the next dose at the regularly scheduled time. The patient should be told not to take two doses at the same time if a dose has been missed.
Elderly
No dose adjustment is required in patients over the age of 65 years (see section 5.2). There is limited clinical experience in patients over the age of 75 years. Therefore Opsumit should be used with caution in this population (see section 4.4).
Hepatic impairment
Based on pharmacokinetic (PK) data, no dose adjustment is required in patients with mild, moderate or severe hepatic impairment (see sections 4.4 and 5.2). However, there is no clinical experience with the use of macitentan in PAH patients with moderate or severe hepatic impairment. Opsumit must not be initiated in patients with severe hepatic impairment, or clinically significant elevated hepatic aminotransferases (greater than 3 times the Upper Limit of Normal (> 3 × ULN); see sections 4.3 and 4.4).
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