FDA批准首个突破性治疗认定药品
出自识林
FDA批准首个突破性治疗认定药品
笔记 2013-11-05 FDA 临床试验人数的多少，并非突破性治疗药品的热点。Gazyva的重要性在于，是首个真正意义上通过这一通道获批的药品。见下文。 FDA批准首个突破性治疗认定药品 2013年11月1日，FDA宣布首个通过突破性治疗药品通道审评的药品获批，这一新的审评通道旨在加速审评流程，使非常有前途的产品进入市场。罗氏公司的Gazyva (obinutuzumab)成为通过这一审批通道获批的首个药品。 根据2012年《FDA安全与创新法案》（FDASIA），授权FDA使用突破性疗法治疗药品认定这一加速审评通道。突破性疗法药品指依据初步临床证据，显示药品在临床上有意义的终点方面，比现有疗法有确实改善，拟用于治疗严重病症。 与快速通道认定、优先审评认定或加速审批不同，突破性治疗药品认定并不意味着建立在缩短时间表或替代标记物基础之上的加快研发，而是通过加强诸如具备快速通道认定的所有特征的审评工具应用，并由FDA对药品研发强化指导。 2013年1月，FDA接受了来自Vertex制药公司的两份突破性治疗药物认定申请，包括治疗囊性纤维化的Kalydeco(ivacaftor)适应症扩展（已获批），以及Kalydeco与一种处于试验阶段的化合物的复方制剂。这是FDA接受的首份突破性治疗药品申请。 2013年11月1日，罗氏公司的Gazyva (obinutuzumab)通过突破性治疗药品认定通道获批，这是首个真正意义上通过这一通道获批的药品。Gazyva用于与苯丁酸氮芥联用治疗慢性淋巴细胞白血病。由于该药品在美国的目标使用人群少于20万，同时认定为罕见病用药。罗氏公司与2013年7月递交生物制品许可申请，同时被给予优先审评。 识林-Kapok 2013-11-02 FDA approves Gazyva for chronic lymphocytic leukemia The U.S. Food and Drug Administration today approved Gazyva (obinutuzumab) for use in combination with chlorambucil to treat patients with previously untreated chronic lymphocytic leukemia (CLL). CLL is a blood and bone marrow disease that usually gets worse slowly. According to the National Cancer Institute, 15,680 Americans will be diagnosed and 4,580 will die from the disease this year. Gazyva works by helping certain cells in the immune system attack cancer cells. Gazyva is intended to be used with chlorambucil, another drug used to treat patients with CLL. Gazyva is the first drug with breakthrough therapy designation to receive FDA approval. This designation was requested by the sponsor and granted soon after the biologic license application to support marketing approval was submitted to the FDA. The FDA can designate a drug a breakthrough therapy at the request of the sponsor if preliminary clinical evidence indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases. The FDA also granted Gazyva priority review because the drug demonstrated the potential to be a significant improvement in safety or effectiveness in the treatment of a serious condition. And the FDA granted Gazyva orphan product designation because it is intended to treat a rare disease. “Today's approval represents an important new addition to the treatments for patients with CLL,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval reflects the promise of the Breakthrough Therapy Designation program, allowing us to work collaboratively with companies to expedite the development, review and availability of important new drugs.” Gazyva's approval for CLL is based on a study of 356 participants in a randomized open-label multicenter trial comparing Gazyva in combination with chlorambucil to chlorambucil alone in participants with previously untreated CLL. Participants receiving Gazyva in combination with chlorambucil demonstrated a significant improvement in progression free survival: an average of 23 months compared with 11.1 months with chlorambucil alone. The most common side effects observed in participants receiving Gazyva in combination with chlorambucil were infusion-related reactions, a decrease in infection-fighting white blood cells (neutropenia), a low level of platelets in the blood (thrombocytopenia), low red blood cells (anemia), pain in the muscles and bones (musculoskeletal pain), and fever (pyrexia). Gazyva is being approved with a boxed warning regarding Hepatitis B virus reactivation and a rare disorder that damages the material that covers and protects nerves in the white matter of the brain (progressive multifocal leukoencephalopathy). These are known risks with other monoclonal antibodies in this class and rare cases were identified in participants on other trials of Gazyva. Patients should be advised of these risks and assessed for Hepatitis B virus and reactivation risk. Gazyva is marketed by Genentech, a member of the Roche Group, based in South San Francisco, Calif. 原文请见 FDA approves Gazyva for chronic lymphocytic leukemia 岗位必读建议：
文件适用范围： 文件要点总结：
以上仅为部分要点，请阅读原文，深入理解监管要求。 |