FDA Approves First Pegfilgrastim Biosimilar, Fulphila
最近ASCO比较火，把这条新闻掩盖了。
1 正文
2 CRL
3 补充
4 其他的事情
1 正文
FDA批准了首个培非格司亭的生物类似药：pegfilgrastim-jmdb(商品名 Fulphila)，这是首个在美获批的培非格司亭的生物类似药，由Mylan和Biocon联合申报。用于降低非骨髓性恶性肿瘤患者使用具有骨髓抑制作用的药物化疗导致发热性嗜中性白血球减少引发的感染危险(发热、白细胞异常减少等)。
The U.S. Food and Drug Administration today approved Fulphila (pegfilgrastim-jmdb) as the first biosimilar to Neulasta (pegfilgrastim) to decrease the chance of infection as suggested by febrile neutropenia (fever, often with other signs of infection, associated with an abnormally low number of infection-fighting white blood cells), in patients with non-myeloid (non-bone marrow) cancer who are receiving myelosuppressive chemotherapy that has a clinically significant incidence of febrile neutropenia.
戈大大说：1）把新的类似药带给患者是FDA的最优先考虑的，也是促进竞争、降低开支、提高可及性的重要手段；2）还会继续优先审评，提高上市效率+符合严格标准；3）夏天还有新政策来促进类似药发展；4）生物制品还是贵，发展生物类似药可以快速有效让患者享受竞争带来的红利。
“Bringing new biosimilars to patients is a top priority for the FDA, and a key part of our efforts to help promote competition that can reduce drug costs and promote access,” said FDA Commissioner Scott Gottlieb, M.D. “We’ll continue to prioritize reviews of these products to help ensure that biosimilar medications are brought to the market efficiently and through a process that makes certain that these new medicines meet the FDA’s rigorous standard for approval. This summer, we’ll release a comprehensive new plan to advance new policy efforts that promote biosimilar product development. Biologics represent some of the most clinically important, but also costliest products that patients use to promote their health. We want to make sure that the pathway for developing biosimilar versions of approved biologics is efficient and effective, so that patients benefit from competition to existing biologics once lawful intellectual property has lapsed on these products.”
Biological products are generally derived from a living organism and can come from many sources, such as humans, animals, microorganisms or yeast. A biosimilar is a biological product that is approved based on data showing that it is highly similar to a biological product already approved by the FDA (reference product) and has no clinically meaningful differences in terms of safety, purity and potency (i.e., safety and effectiveness) from the reference product, in addition to meeting other criteria specified by law.
中心就一个，和之前批的类似药一样，只是biosimilar，不是interchangeable product。
The FDA’s approval of Fulphila is based on review of evidence that included extensive structural and functional characterization, animal study data, human pharmacokinetic and pharmacodynamic data, clinical immunogenicity data, and other clinical safety and effectiveness data that demonstrates Fulphila is biosimilar to Neulasta. Fulphila has been approved as a biosimilar, not as an interchangeable product.
常见副作用骨痛、肢痛，对G-CSF类产品严重过敏的不适用Fulphila。
The most common side effects of Fulphila are bone pain and pain in extremities. Patients with a history of serious allergic reactions to human granulocyte colony-stimulating factors such as pegfilgrastim or filgrastim products should not take Fulphila.
严重的副作用包括：脾脏破裂、急性呼吸窘迫综合症ARDS、严重过敏（过敏反应、肾脏急性炎症(血管球性肾炎)、白细胞异常升高）、毛细血管渗漏综合征、可能肿瘤生长，致死性镰状细胞危象也有发生。
Serious side effects from treatment with Fulphila include rupture of the spleen, acute respiratory distress syndrome, serious allergic reactions including anaphylaxis, acute inflammation of the kidney (glomerulonephritis), an abnormally high level of white blood cells (leukocytosis), capillary leak syndrome and the potential for tumor growth. Fatal sickle cell crises have occurred.
法国监管机构检查了Biocon的车间后提了一堆问题，因此公司已经撤回了欧盟的申报，后面会再次提交申请。
The company also withdrew its application for the product from the European Medicines Agency following a French regulatory inspection noted 35 problems with Biocon’s manufacturing facility. It later resubmitted the application to the EMA, where it awaits a regulatory decision.
Mylan和Biocon之前已经有获得FDA批准的生物类似药：即曲妥珠单抗的类似药，商品名Ogivri，但还没进入市场销售。
The Mylan–Biocon partnership currently has 1 additional biosimilar product approved by the FDA: Ogivri, a trastuzumab biosimilar referencing Herceptin. The drug has not yet been launched in the United States.
2 CRL
17年10月也是收到CRL，但是主要还是车间改造后的重新申报，与药本身的数据无关。
3 补充：3期临床
4 其他的事情
关于公众号的：现在有几个志同道合的人：小和尚、南院和凿冰室主人，下半年可能有个看器械的朋友进来，一直在考虑这个公众号的内容，首先是良机，肯定不会刻意写大标题；然后还是交流的心态，所以从下周开始除了每周二、四、六的更新，一个月会出1-2篇万字以上的文字：可能是读书笔记，也可能是总结之类。总的来说会继续努力更新维护好，但是我们几个也考虑到自身水平有限，难免很多错误，还是恳请读者谅解。最后谢谢大家关注捧场。
魔都的八卦小和尚
帝都的南院枢密使
西川的凿冰室主人