强生淋巴瘤药物Imbruvia获FDA批准

强生（JNJ）和Pharmacyclics 11月13日宣布，FDA已批准血癌药物ibrutinib（拟用商品名为Imbruvica），用于既往已接受至少一种其他药物（如Celgene公司的Revlimid）治疗的套细胞淋巴瘤（mantle cell lymphoma，MCL）成人患者的治疗。该药成为获得FDA突破疗法认定并获批的第2个药物，ibrutinib将成为上市的首个BTK靶向药物。

FDA于本月早些时候批准了罗氏（Roche）的Gazyva，用于治疗慢性淋巴细胞白血病（CLL），是首个获FDA突破疗法认定并获批的药物。

在美国，套细胞淋巴瘤（MCL）占到了非霍奇金淋巴瘤病例的6%，该病在确诊时，通常已扩散至淋巴结、骨骼和其他器官。

目前，ibrutinib也正在等待FDA批准用于CLL。一些分析人士原以为，FDA会同时批准ibrutinib用于MCL和CLL。

RBC资本市场分析师Michael Yee预计，ibrutinib的长期年度全球销售额将达到50亿美元。

Ibrutinib由杨森与Pharmacyclics合作开发，目前正调查用于数种类型血癌的治疗。

强生分别于今年7月10日和10月30日向FDA和欧洲药品管理局（EMA）提交了Ibrutinib的上市许可申请（MAA），寻求批准用于复发性或难治性慢性淋巴细胞白血病（CLL）/小淋巴细胞白血病（SLL）或复发性或难治性套细胞淋巴瘤（MCL）成人患者的治疗。

CLL/SLL和MCL属于一组名为B细胞恶性肿瘤的血液癌症。许多患者在治疗后会复发，可能在治疗过程中使用数种药物。

关于Ibrutinib：

Ibrutinib是一种名为Bruton酪氨酸激酶（BTK）抑制剂的首创新药。BTK是一个重要的蛋白，参与介导调控B细胞成熟和生存的胞内信号通路。在恶性B细胞中，B细胞受体信号通路过度活跃，该信号通路即包括BTK。

Ibrutinib能够与BTK形成强有力的共价键，从而抑制恶性B细胞中过度活跃的细胞生存信号的传输。

英文原文：U.S. health regulators approve J&J, Pharmacyclics lymphoma drug

Nov 13 (Reuters) - U.S. health regulators on Wednesday approved a drug to treat a rare and aggressive form of non-Hodgkin lymphoma developed by Johnson & Johnson and Pharmacyclics Inc, becoming the second drug that had received the FDA's new breakthrough therapy designation to gain approval.

The drug, to be sold under the brand name Imbruvica, and known chemically as ibrutinib, was approved to treat mantle cell lymphoma in patients who have received prior treatment with at least one other medicine, such as Celgene Corp's Revlimid. It works by inhibiting an enzyme needed by the cancer to multiply and spread.

Mantle cell lymphoma, which represents about 6 percent of non-Hodgkin lymphoma cases in the United States, has typically spread to lymph nodes, bone marrow and other organs by the time it is diagnosed, the Food and Drug Administration said.

The FDA earlier this month approved Roche's Gazyva to treat chronic lymphocytic leukemia (CLL), another type of blood cancer, making it the first drug approved after receiving the breakthrough designation.

The FDA gives a breakthrough designation when it believes a medicine may offer a substantial improvement over existing therapies for serious or life-threatening diseases.

"The agency worked cooperatively with the companies to expedite the drug's development, review and approval, reflecting the promise of the breakthrough therapy designation program," Richard Pazdur, director of the FDA's Office of Hematology and Oncology Products, said in a statement.

Imbruvica is also awaiting an approval decision to treat CLL, which some analysts believed would come at the same time as the U.S. lymphoma approval.

"This is a relative short-time disappointment versus Wall Street expectations for simultaneous approval in both indications with a broad label," RBC Capital Markets analyst Michael Yee said in a research note.

Yee, in an email, said RBC is forecasting long-term annual worldwide sales of $5 billion for Imbruvica.

Pharmacyclics shares were up $3.20, or 2.6 percent, at $122.87 on Nasdaq. Shares of Johnson & Johnson were down 85 cents, or 0.9 percent ,at $92.71 on the New York Stock Exchange.