Scott Lesley J
Springer, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Drugs. 2016 Mar;76(3):413-8. doi: 10.1007/s40265-016-0549-4.
Selexipag (Uptravi(®)) is a highly selective, long-acting, nonprostanoid, prostacyclin receptor agonist that is being developed by Actelion Pharmaceuticals Ltd and Nippon Shinyaku. Oral selexipag is approved in the USA for the treatment of pulmonary arterial hypertension (PAH; WHO Group I) to delay disease progression and reduce the risk of hospitalization for PAH. It has subsequently been approved in Canada for the long-term treatment of PAH, and received a positive opinion in the EU for the treatment of PAH in adult patients with WHO functional class II-III. Selexipag received orphan drug designation for the treatment of PAH in Japan in 2014 and is in undergoing regulatory review in several countries for use in this indication. In the large, event-driven, phase III GRIPHON trial, selexipag reduced the risk of the primary composite endpoint of death or a complication related to PAH (whichever occurred first) by 40 % compared with placebo in patients with PAH (80 % were also receiving stable dosages of an endothelin receptor antagonist and/or a phosphodiesterase 5 inhibitor). This article summarizes the milestones in the development of selexipag leading to this first approval for PAH.
司来帕格（Uptravi(®)）是一种高选择性、长效、非前列腺素类前列环素受体激动剂，由Actelion制药有限公司和日本新药株式会社共同研发。司来帕格口服制剂在美国已获批准，用于治疗肺动脉高压（PAH；WHO第I组），以延缓疾病进展并降低PAH患者住院风险。随后，它在加拿大获批用于PAH的长期治疗，并在欧盟获得积极意见，用于治疗WHO功能分级为II - III级的成年PAH患者。司来帕格于2014年在日本获得孤儿药认定，用于治疗PAH，目前正在多个国家接受该适应症的监管审查。在大型、事件驱动的III期GRIPHON试验中，与安慰剂相比，司来帕格使PAH患者死亡或与PAH相关并发症（以先发生者为准）这一主要复合终点的风险降低了40%（80%的患者同时还接受稳定剂量的内皮素受体拮抗剂和/或磷酸二酯酶5抑制剂）。本文总结了司来帕格在PAH首次获批之前的研发历程中的重要节点。
