201年1月20日讯 /生物谷BIOON/ --近日，勃林格殷格翰（Boehringer Ingelheim）罕见病药物Ofev（nintedanib）在欧盟监管方面收获大好消息，欧盟委员会（EC）已批准口服药物Ofev用于特发性肺纤维化（IPF）的治疗。
在美国，Ofev（nintedanib）于2014年10月15日获FDA批准，同一天获批的还有另一款药物Esbriet（比非尼酮），Esbriet和Ofev的上市，结束了美国IPF患者群体无药可用的局面。Esbriet由InterMune开发，罗氏于2014年8月耗资83亿美元收购InterMune后将该药收入囊中，该笔交易在很大程度上就是因为罗氏看中了Esbriet在特发性肺纤维化（IPF）市场中的前景。
在欧盟，Esbriet于2011年获欧盟委员会（EC）批准。此次Ofev拿下欧盟批文，也标志着，在欧洲市场，勃林格将与罗氏一决雌雄！
特发性肺纤维化（IPF）是一种罕见的肺部疾病，根据肺纤维化联盟（CPF）数据，每年IPF死亡病例达40000例。分析师预计，IPF药物市场的年销售将超过20亿美元。
Ofev在欧盟的获批，是基于III期INPULSIS项目的数据，该项目包括2项III期研究（INPULSIS-1和INPULSIS-2），涉及来自24个国家的1066例特发性肺纤维化（IPF）患者，研究结果显示，在广泛类型IPF群体中，nintedanib显著延缓了病情的恶化，并显著降低了急性发作风险，2项研究均达到了主要终点。
Nintedanib是一种口服三联血管激酶抑制剂，可同时阻断3种生长因子受体：血管内皮生长因子受体（VEGFR 1-3）、血小板源性生长因子受体（PDGFR α和β）、成纤维细胞生长因子受体（FGFR 1-3）。所有这3种受体在血管生成和肿瘤生长过程中均发挥着重要作用。这些受体的阻断，可能导致血管生成的抑制，而血管生成在肿瘤生长中起着关键作用。
目前，勃林格殷格翰正在多种实体瘤中评价nintedanib，其中晚期非小细胞肺癌（NSCLC）、结直肠癌、卵巢癌已处于III期临床，间皮瘤、肾癌（肾细胞癌）、肝癌（肝细胞癌）处于II期临床。
英文原文：OFEV (nintedanib) approved in the EU for the treatment of IPF
-Nintedanib* slows disease progression by reducing the decline in lung function by 50% in a broad range of IPF patient types**
-Nintedanib* significantly reduced the risk of adjudicated acute exacerbations? by 68%
-EC approval is based on results from the replicate Phase III INPULSIS? trials, involving 1,066 patients from 24 countries
Ingelheim, Germany, 19 January 2015 – Boehringer Ingelheim today announced that the European Commission (EC) has approved nintedanib* for the treatment of idiopathic pulmonary fibrosis (IPF), following an expedited review and positive CHMP opinion on 20 November 2014. Nintedanib* will be marketed in the EU under the brand name OFEV?. IPF is a debilitating and fatal lung disease – with a median survival of 2-3 years after diagnosis.
“Approval of this treatment for patients in the EU is a significant step towards meeting the substantial unmet need in IPF. Patients suffering from this chronic, debilitating disease can now be offered a new treatment option that has been shown to have a clinically meaningful effect on their disease,” said Professor Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim. “This approval is another milestone in Boehringer Ingelheim’s ongoing efforts with regard to innovation in rare diseases in general and our continuing research for the benefit of patients affected by such a dreadful disease as IPF in particular.”
EC approval is based on results from the replicate Phase III INPULSIS? trials, involving 1,066 patients from 24 countries. INPULSIS? results showed that nintedanib* slowed disease progression by reducing the annual rate of decline in lung function by 50% in a broad range of IPF patient types including patients with early disease (forced vital capacity (FVC) >90% pred), limited radiographic fibrosis (no honeycombing) on high resolution computed tomography (HRCT) and those with emphysema.2 Nintedanib*, only one capsule twice a day, is the first targeted treatment for IPF to consistently meet the primary endpoint in two identically designed Phase III trials. Nintedanib* has been shown to significantly reduce the risk of adjudicated acute exacerbations?
by 68%.2
Study investigator Professor Luca Richeldi, Professor of Respiratory Medicine, Chair of Interstitial Lung Disease at the University of Southampton, United Kingdom, said “Until recently, treatment options for patients with IPF were limited. The approval of nintedanib* in the EU gives patients with a life threatening illness a choice of therapy with proven efficacy. Clinical data demonstrate that nintedanib* reduces the annual decline of lung function by approximately half. Data also showed that nintedanib* reduced the risk of acute exacerbations, which can lead to hospitalisation and death.”
Worldwide IPF affects as many as 14–43 people per 100,000.3,4 It most commonly affects people over the age of 50.5 IPF causes permanent scarring of the lungs and decreases the amount of oxygen the lungs can supply to major organs of the body.6,7
“It’s great that there is now a choice of treatments for IPF patients. This approval provides important hope for patients and caregivers living with this awful disease,” said Dr Toby Maher, Consultant Respiratory Physician at the Royal Brompton Hospital in London, United Kingdom.