FDA核准:Cellcept、Prochymal、志贺毒素抗体


  Jan. 9, 2006 - FDA以孤儿药核准mycophenolate mofetil用于治疗重症肌无力;一种成人干细胞产品,治疗急性排斥反应;以及2种治疗产生志贺毒素大肠杆菌感染的单株抗体。
  
  孤儿药Mycophenolate Mofetil(Cellcept)用于治疗重症肌无力
  1月3日,FDA以孤儿药物核准免疫抑制药物mycophenolate mofetil(Cellcept,由Aspreva以及罗氏药厂制造),用于治疗重症肌无力(MG)。
  
  MG是一种失能性、慢性的自体免疫神经肌肉疾病,可能会影响任何自主肌肉,但是通常牵涉到控制眼睛运动、咀嚼、与吞咽、咳嗽、与面部表情的肌肉。
  
  目前的疗法包括胆碱酯抑制剂、类固醇、与其它免疫抑制剂,包括azathioprine;使用这些药物很少使疾病完全消退,而且通常需要长期服用免疫抑制剂。
  
  根据该公司新闻稿,一项随机分派、双盲、安慰剂控制全球性第三期临床试验正在进行中,这项试验将会检验该药物使用于治疗MG为期36周,与低剂量类固醇并用以维持或是改善症状控制的疗效与安全性。
  
  这项试验的试验终点为最低及病活性与低类固醇剂量,试验结果将于2006年底发表。
  
  Mycophenolate之前由FDA与世界各地其它官方单位核准与cyclosporine及类固醇并用于预防成人接受异源性肾脏、心脏、或是肝脏移植的器官排斥反应;在某些国家,该药物也被核准于预防孩童肾脏移植排斥。
  
  孤儿药成人干细胞产品(Prochymal)使用于GVHD
  2005年12月20日,FDA以孤儿药核准一种人类干细胞产品(Prochymal,由Osiris药厂制造),使用于治疗急性排斥反应(GVHD),该产品之前于2005年以快速追踪方式加速审查。
  
  GVHD是一种可能致命的免疫排斥反应,影响约50%接受异源性血液生成干细胞移植治疗白血病与淋巴癌等癌症的病患;在许多病例中,急性GVHD对类固醇的治疗反应不佳,而目前而言,免疫抑制剂是治疗的主力。
  
  根据该公司新闻稿,该产品是一种人类间叶干细胞剂型,可以透过2种机转调控发炎反应,分别是降低抗发炎细胞激素的产生及增加抗发炎细胞激素的浓度;也会降低T细胞复制速率。
  
  除此之外,干细胞已经表现出可以迁移到发炎与组织受损部位的能力,在那里这些细胞会加速复原速度;对GVHD病患而言,使用这种产品可以减少腹泻、改善细胞层内里溃疡的复元、以及修复皮肤病灶。
  
  该公司目前正于2项第2期、安慰剂控制临床试验收纳病患,预计评估2种不同剂量使用的类固醇治疗于急性与严重急性GVHD病患之疗效与安全性。
  
  该产品也正在研究使用于其它发炎疾病,例如克隆氏症的疗效。
  
  孤儿药志贺毒素抗体用于治疗STEC感染
  FDA于2005年12月以孤儿药核准2种嵌合抗志贺毒素抗体(caStx1与caStx2,由Caprion药厂制造),使用于治疗产生志贺毒素的大肠杆菌(STEC)感染。
  
  根据该公司新闻稿,这种疾病目前并没有治疗方法,这种透过受污染食物或饮水传播的疾病在美国每年有超过10万人受到感染。
  
  该抗体可以中和循环中的志贺毒素(Stx1与Stx2),藉此治疗疾病并且预防严重的并发症,例如肠胃道疾病、血便、红血球与血小板崩解、以及出血性尿毒症候群(HUS)。
  
  该产品目前正在一项寻找剂量的第1期美国临床试验中,使用于STEC感染孩童病患,评估其预防HUS的效果;该产品最近也被欧洲医药评估署以孤儿药方式核准使用于这个适应症。

FDA Approvals: CellCept, Proch

By Yael Waknine
Medscape Medical News

Jan. 9, 2006 — The FDA has approved orphan drug status for mycophenolate mofetil for the treatment of myasthenia gravis; an adult stem cell product for the treatment of acute graft vs host disease; and 2 therapeutic monoclonal antibodies for the treatment of Shiga toxin–producing Escherichia coli infections.


Orphan Drug Mycophenolate Mofetil (CellCept) for Myasthenia Gravis

On January 3, the FDA approved orphan drug status for the immunosuppressant drug mycophenolate mofetil (CellCept, made by Aspreva Pharmaceuticals Corporation and Roche) in the treatment of myasthenia gravis (MG).

MG is a debilitating, chronic autoimmune neuromuscular disease that can affect any voluntary muscle but most frequently involves those controlling eye movements, chewing, swallowing, coughing, and facial expressions.

Current treatments include cholinesterase inhibitors, steroids, and other immunosuppressants such as azathioprine; complete remission is infrequent and long-term immunosuppression is usually required.

According to a company news release, a randomized, double-blind, placebo-controlled global phase 3 study is currently being conducted to evaluate the efficacy and safety of mycophenolate for maintaining or improving symptom control with reduced doses of corticosteroids in patients with MG for 36 weeks.

Primary end points of the trial include both minimal disease activity and low steroid dose. Results are anticipated for late 2006.

Mycophenolate is approved by the FDA and other regulatory authorities worldwide for use in conjunction with cyclosporine and corticosteroids for the prophylaxis of organ rejection in adult patients receiving allogenic renal, cardiac, or hepatic transplant. In some countries, it is also approved for use in pediatric kidney transplantation.


Orphan Drug Adult Stem Cell Product (Prochymal) for GVHD

On December 20, 2005, the FDA approved orphan drug status for an adult stem cell product (Prochymal, made by Osiris Therapeutics, Inc) in the treatment of acute graft-versus-host disease (GVHD). The product was previously granted fast-track status in January 2005.

GVHD is a potentially fatal form of immune rejection that affects approximately 50% of patients who receive an allogenic hematopoietic stem cell transplant for the treatment of cancers such as leukemia and lymphoma. In many cases, acute GVHD is refractory to steroids and immunosuppressive agents that are the current standard of care.

According to a company news release, the product is a formulation of human mesenchymal stem cells that modulates the inflammatory response via 2 mechanisms: decreasing the production of pro-inflammatory cytokines while increasing levels of anti-inflammatory cytokines; and reducing T-cell proliferation rates.

In addition, the stem cells have demonstrated an ability to migrate to sites of inflammation and tissue damage, where they facilitate the repair process. In patients with GVHD, use of the product has reduced diarrhea, improved ulcerations in the intestinal lining, and healed skin lesions.

The company is currently enrolling patients in 2 ongoing phase 2, placebo-controlled clinical trials designed to evaluate product efficacy at 2 dose levels in patients with acute and severe acute GVHD receiving standard steroid therapy.

The product is also being studied for use in other immunologic disease applications such as Crohn's disease.


Orphan Drugs Shiga Toxin Antibodies for STEC Infections

The FDA approved in December 2005 orphan drug status for 2 chimeric anti-Shiga toxin antibodies (caStx1 and caStx2, made by Caprion Pharmaceuticals, Inc) in the treatment of Shiga toxin–producing Escherichia coli (STEC) infections.

According to a company news release, there is no known treatment for the disease, which is transmitted via contaminated food or water and affects more than 100,000 individuals annually in the United States.

The antibodies are intended to neutralize circulating Shiga toxins (Stx1 and Stx2), thereby treating the disease and preventing serious complications such as gastrointestinal disease, bloody diarrhea, destruction of red blood cells and platelets, and hemolytic uremic syndrome (HUS).

The product is currently being evaluated for preventing HUS in a dose-escalating, phase 1 US clinical trial of STEC-infected pediatric patients. It was also recently designated as an orphan drug for this indication by the European Medicines Evaluation Agency.

Reviewed by Gary D. Vogin, MD

    
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