普纳替尼撤市原因分析及对我国新药研发的启示

普纳替尼（ponatinib）是一种多靶点的激酶抑制剂，已经被美国和欧盟批准用于治疗慢性粒细胞白血病及费城染色体阳性的急性淋巴细胞白血病。2013年10月31日，鉴于“危及生命的血栓和血管重度狭窄”风险，FDA要求生产商暂停其销售和推广，这也是首个被撤市的小分子激酶抑制剂类抗肿瘤药。因普纳替尼未来我国申请进口，其撤市不会给我国造成直接影响，但会影响到其他在我国上市或正在研发的同类品种的市场销售或上市进度。因此，建议我国药品监管部门要加强激酶类小分子抗肿瘤药的不良反应事件的监管，并及时将相关信息反馈给国内相应仿制药企业。制药企业在新药上市前要进行充分的安全性评价，尽早发现潜在的风险，以便采取有效措施避免损失。

Cause analysis on withdrawal of ponatinib and its implication for drug development in China

Iclusig is an inhibitor of mutiple kinases indicated for the treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia (CML) or Philadelphia chromosome positive acute lymphoblastic leukemia (Ph⁺ ALL). On October 31, 2013, the U.S. Food and Drug Administration (FDA) has asked the manufacturer of the leukemia chemotherapy drug Iclusig (ponatinib) to suspend marketing and sales of Iclusig because of the risk of life-threatening blood clots and severe narrowing of blood vessels. This is also the first withdrawn small molecule kinase inhibitor of anticancer drugs. The withdrawal of ponatinib does not directly affect Chinese domestic drug market because it was not developed clinical studies and approved. But it will impact the marketing sale or progress of other molecule kinase inhibitor of anticancer drugs including approved and pipeline drugs. Therefore, we suggest that the drug supervision departments should strengthen the monitoring of post-approval drug safety, including small molecule kinase inhibitor of anticancer drugs. The drug supervision departments should feedback drug adverse effects information timely to the relevant corresponding domestic generic drug companies. Pharmaceutical companies should conduct adequate drug safety assessment, early detect the potential risks before new drugs launch, in order to take effective measures to protect patients from drug risks.