FDA批准强生旗下戈利木单抗治疗溃疡性结肠炎新适应症

5月15日，美国食品药品管理局（FDA）批准Simponi（戈利木单抗）注射剂新适应症，用于治疗成人患者中度至重度溃疡性结肠炎。

Simponi通过阻断肿瘤坏死因子(TNF)而发挥作用，TNF在引起异常炎症和免疫应答中扮演着重要的角色。Simponi先前已被批准用于治疗类风湿性关节炎、银屑病关节炎和强直性脊柱炎（影响脊柱和骨盆关节的关节炎），现在这个药物被批准用于治疗对先前治疗产生耐药(难治性)或需要持续类固醇治疗的中度至重度溃疡性结肠炎成人患者。

溃疡性结肠炎是一种慢性疾病，在美国大约有62万人受此疾病的影响。这种疾病能在大肠内引起炎症和溃疡，是两种主要的慢性炎症性肠病之一。这种炎症能导致腹部不适、胃肠出血、产生脓和腹泻。

FDA药品评价与研究中心肠胃和先天缺陷产品部门副主任Andrew E. Mulberg医学博士说：“Simponi对于中度至重度溃疡性结肠炎患者是一种重要的新治疗选择，患有严重和痛苦溃疡性结肠炎症状的患者有一种额外的治疗选择是至关重要的，因为患者要经受疾病的影响，并且对治疗的响应不同。”

Simponi用于溃疡性结肠炎的安全性和有效性基于两项临床研究。患者评价包括排便频次检测、直肠出血、内窥镜结果和医生的综合评估。

在第一项研究中，513名不能忍受或对其它治疗不响应的中度至重度溃疡性结肠炎患者被随机配给Simponi或安慰剂。结果显示，6周之后与安慰剂组相比，更大比例的Simponi治疗患者获得临床应答和临床缓解，通过内窥镜检查患者的直肠外观已有改善。

在第二项研究中，310名对Simponi响应的中度至重度溃疡性结肠炎患者被随机配给Simponi或安慰剂。与安慰剂组患者相比，更大比例的Simponi治疗患者在54周内保持了临床应答，在第30周和第54周都有临床缓解，通过内窥镜检查其直肠外观在第30周和第54周均已有改善。

Simponi治疗患者最常见的副作用有上呼吸道感染和注射部位红肿。Simponi治疗患者出现严重感染、侵入性真菌感染、乙型肝炎感染重新激活、淋巴瘤、心脏衰竭、神经系统紊乱和过敏反应的风险升高。

Simponi由强生旗下的Janssen Ortho 生物科技公司上市销售。

FDA approves Simponi to treat ulcerative colitis
The U.S. Food and Drug Administration today approved a new use for Simponi (golimumab) injection to treat adults with moderate to severe ulcerative colitis.
Simponi works by blocking tumor necrosis factor (TNF), which plays an important role in causing abnormal inflammatory and immune responses. Previously approved to treat rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis (arthritis affecting the joints in the spine and the pelvis), Simponi is now approved to treat adults with moderate to severe ulcerative colitis that is resistant (refractory) to prior treatment or requires continuous steroid therapy.
Ulcerative colitis is a chronic disease that affects about 620,000 Americans. It causes inflammation and ulcers in the inner lining of the large intestine and is one of two main forms of chronic inflammatory bowel disease. The inflammation can lead to abdominal discomfort, gastrointestinal bleeding, production of pus and diarrhea.
“Simponi is an important new treatment option for patients with moderate to severe ulcerative colitis,” said Andrew E. Mulberg, M.D., deputy director of the Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research. “It is critical that patients suffering from the serious and painful symptoms of ulcerative colitis have additional treatment options since patients experience the effects of the disease and respond to treatments differently.”
The safety and effectiveness of Simponi for ulcerative colitis were established in two clinical studies. Evaluations of patients included measures of stool frequency, rectal bleeding, endoscopic findings and a physician’s overall assessment.
In the first study, 513 patients with moderate to severe ulcerative colitis who could not tolerate or failed to respond to other therapies were randomly assigned to receive Simponi or a placebo. Results showed that a greater proportion of Simponi-treated patients achieved clinical response, clinical remission and, as seen during endoscopy, had improved appearance of the colon after six weeks compared with the placebo group.
In the second study, 310 patients with moderate to severe ulcerative colitis who were responders to Simponi were randomly assigned to receive Simponi or placebo. A greater proportion of Simponi-treated patients maintained clinical response through week 54 and had clinical remission at both weeks 30 and 54...