亚盛医药-B(06855.HK)：亚盛医药核心品种HQP1351(奥雷巴替尼)获欧盟委员会孤儿药资格认定，用于治疗慢性髓细胞白血病
格隆汇11月22日丨亚盛医药-B(06855.HK)公吿，欧盟委员会(EC)日前授予公司1类新药奥雷巴替尼(Olverembatinib，HQP1351，曾用名耐克替尼、奥瑞巴替尼)孤儿药资格认定，用于治疗慢性髓细胞白血病(CML)。这是亚盛医药的药物品种在欧盟获得的首项孤儿药资格认定，也是该在研药物继获美国食品和药品监督管理局(FDA)孤儿药资格认定后的第二项孤儿药认证。
"孤儿药"又被称为罕见药，指用于预防、治疗、诊断罕见病的药品。在欧盟，孤儿药资格认定由欧盟委员会(EC)根据欧洲药品管理局(EMA)孤儿药委员会(COMP)的积极意见授予。只有用于治疗在欧盟发病率低于5/10000、危及生命或严重影响生存质量、且治疗领域存在巨大未被满足需求的罕见疾病的药物，才能在欧盟被授予孤儿药资格认定。
而获此认定的药物必须通过足够的非临床和临床数据表明其能够带来比现有已获批疗法更大的获益。为鼓励罕见病药品的研发，欧盟提供了一系列激励措施。奥雷巴替尼获此项孤儿药认定，将有助于该药物在欧盟的后续研发和商业化开展等方面享受积极的政策支持，包括临床研究计划援助、相关费用减免、特别是在批准上市后可享有10年市场独占权。
CML是一种罕见的恶性血液疾病，在欧盟27国的年发病率为2.43/100001。随着靶向BCR-ABL的酪氨酸激酶抑制剂(TKI)的上市，CML的治疗方式得以革新。尽管第一代和第二代药物BCR-ABLTKI对CML的治疗具有显著的临床效益，但获得性耐药一直是CML治疗的主要挑战。BCR-ABL激酶区突变是获得性耐药的重要机制一，其中T315I突变是常见的耐药突变类型一，在耐药CML中的发生率可达25%左右。伴有T315I突变的CML患者对目前所有一代、二代BCR-ABL抑制剂均耐药，因此临床上急需可有效治疗T315I突变CML的第三代BCR-ABL抑制剂。
奥雷巴替尼是亚盛医药原创1类新药，是新型的第三代BCR-ABLTKI，用于治疗对一代、二代TKI耐药的CML，特别是对T315I突变的CML患者在临床试验中显现良好的疗效。目前，该品种在中国的新药上市申请(NDA)已在审批过程中，用于治疗伴有T315I突变的CML慢性期(CML-CP)及加速期(CML-AP)患者，极有望成为中国首个、全球第二个获批上市的第三代BCR-ABLTKI。奥雷巴替尼还获得中国国家药品监督管理局(NMPA)新药审评中心(CDE)纳入突破性治疗品种，拟治疗一代和二代TKI耐药和╱或不耐受的CML-CP患者。而在美国，该品种已获得美国FDA授予的孤儿药资格和审评快速通道资格；此外，奥雷巴替尼的临床试验进展自2018年开始，连续四年入选美国血液学会(ASH)年会口头报吿，并荣获2019ASH年会"最佳研究"的提名。
Follow us
Find us on
Facebook,
Twitter ,
Instagram, and
YouTube or frequent updates on all things investing.Have a financial topic you would like to discuss? Head over to the
uSMART Community to share your thoughts and insights about the market! Click the picture below to download and explore uSMART app！
Disclaimers
uSmart Securities Limited (“uSmart”) is based on its internal research and public third party information in preparation of this article. Although uSmart uses its best endeavours to ensure the content of this article is accurate, uSmart does not guarantee the accuracy, timeliness or completeness of the information of this article and is not responsible for any views/opinions/comments in this article. Opinions, forecasts and estimations reflect uSmart’s assessment as of the date of this article and are subject to change. uSmart has no obligation to notify you or anyone of any such changes. You must make independent analysis and judgment on any matters involved in this article. uSmart and any directors, officers, employees or agents of uSmart will not be liable for any loss or damage suffered by any person in reliance on any representation or omission in the content of this article. The content of this article is for reference only. It does not constitute an offer, solicitation, recommendation, opinion or guarantee of any securities, financial products or instruments.The content of the article is for reference only and does not constitute any offer, solicitation, recommendation, opinion or guarantee of any securities, virtual assets, financial products or instruments. Regulatory authorities may restrict the trading of virtual asset-related ETFs to only investors who meet specified requirements.
Investment involves risks and the value and income from securities may rise or fall. Past performance is not indicative of future performance.
