2012年9月5日，日本最大的制药商——武田（Takeda）制药，与其合作伙伴NPS制药公司宣布，药物Revestive（teduglutide）上市许可申请（MAA）已获欧盟委员会批准，作为日服一次的药物用于短肠综合征（short bowel syndrome，SBS）成人患者的治疗。
在今年6月21日，Revestive的上市许可申请（MAA）已获欧洲药品管理局（EMA）人用医药产品委员会（CHMP）积极意见。
最初将通过NPP项目（Named Patient Program）向欧洲的患者提供Revestive。
“短肠综合征（SBS）患者遭受营养不良和腹泻，并经常需要胃肠外营养支持来维持生命，”丹麦哥本哈根大学医学院消化内科Palle Bekker Jeppesen医师说道。“对于我们的患者来说，Revestive是一种新颖、独特、重要的治疗选择，同时为有限的治疗医疗设备增加了重要的价值。”
欧洲获批的首个SBS药物
“Teduglutide是欧洲首个获批用于SBS的药物，为这一患者群体提供了一个新的、重要的治疗选择，”武田欧洲及加拿大商业运作中心主任Trevor Smith说道。
“Teduglutide欧洲上市申请的获批对于SBS患者来说是个大好消息，”NPS制药总裁兼CEO Francois Nader说道。“我们期待着与我们的合作伙伴武田一同协作，在欧洲市场推出这一重要产品。”
Revestive的上市许可申请在欧盟成员国有效，预计冰岛及挪威的监管申请将在30天内获批。
此次获批，是基于一项关键性双盲、安慰剂对照的III期安全性及有效性试验，试验中的SBS患者均需要胃肠外营养支持。在试验中，43例患者接受皮下注射0.05mg/kg/day剂量的teduglutide，另外43例患者注射安慰剂，整个治疗周期为24周。
Teduglutide治疗组，在20周及24周时胃肠外营养支持减少20%~100%的患者比例，明显高于安慰剂组（60% vs 30%，p=0.002）。在24周内，teduglutide治疗患者胃肠外营养支持需求量减少达4.4升/周，而安慰剂组这一数据为2.2升/周。Teduglutide治疗组有21例患者取得了至少1天胃肠外应用支持的减少（54%），而安慰剂组仅为9例（23%）。研究中，在剂量、频率及持续方面，teduglutide均具有良好的耐受性。
Revestive 5 mg powder and solvent for injection
In the EU, Revestive® 5 mg powder and solvent for injection is approved for the treatment of adult patients with Short Bowel Syndrome; patients should be stable following a period of intestinal adaptation after surgery.
Expanded Access
Aside from a clinical trial, a drug typically may not be distributed prior to its approval by government regulators, such as the US Food and Drug Administration or European Medicines Agency. However, there are rare exceptions for select patients with serious or immediately life-threatening diseases who have no comparable or suitable treatment alternatives.
When this situation occurs in the US, FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an individual patient, or for intermediate-size groups of patients with similar treatment needs who otherwise do not qualify to participate in a clinical trial2. Similarly, in the EU, Named Patient (or Compassionate Use) Programs create access to particular treatments prior to marketing authorization for patients with life-threatening, chronic or seriously disabling conditions. Compassionate use programs are coordinated and implemented by the EU Member States, which decide independently how and when to open such programs according to national rules and legislation.
Abbreviated SPC
Revestive 5 mg powder and solvent for solution for injection
Summary of Product Characteristics (SPC) is available here.
Therapeutic indications
Revestive® (teduglutide) is indicated for the treatment of adult patients with Short Bowel Syndrome. Patients should be stable following a period of intestinal adaptation after surgery.
Important Safety Information
Posology
Adults: The recommended dose of teduglutide is 0.05 mg/kg body weight once daily.
Special populations
Elderly: No dose adjustment is necessary in patients above the age of 65 years. Renal Impairment: No dose adjustment is necessary for patients with mild renal impairment. In patients with creatinine clearance less than 50 mL/min, the daily dose should be reduced by 50%.
Hepatic Impairment: No dose adjustment is necessary for patients with mild and moderate hep |
