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FDA批准百特Rixubis用于B型血友病儿科患者

来源:生物谷 2014-09-16 09:18

 

2014年9月16日讯 /生物谷BIOON/ --百特(Baxter)9月15日宣布,FDA已批准Rixubis(重组凝血因子IX)用于B型血友病儿科患者的常规预防性治疗、出血控制及围术期管理。此前,FDA已于2013年6月批准Rixubis用于B型血友病成人患者的常规预防、出血控制及围术期管理,该药是获批的首个重组因子IX(rFIX),也是过去15年来首个治疗B型血友病的新疗法。百特于2013年12月向FDA和欧盟提交了Rixubis用于B型血友病儿科患者的上市申请。

Rixubis儿科适应症的获批,是基于一项II/III期儿科临床试验的数据,该试验调查了Rixubis用于治疗23例既往已接受治疗的、12岁及以下中度或重度B型血友病儿科患者时的疗效和安全性。该项研究中,年平均出血率(ABR)为2.0(自发出血和关节出血为0.0),39.1%的患者未经历出血,88.5%的患者经1-2次输液治疗。在止血疗效中,Rixubis在96%的所有出血事件中止血疗效被评为优秀或良好。研究中,无患者产生FIX抑制性抗体。

在儿科B型血有病患者群体中取得的这些积极数据,与此前关键性试验中Rixubis在B型血有病成人患者群体中所观察到的积极数据一致。

Rixubis是一种重组凝血因子IX(rFIX),可用于B型血有病成人患者的常规预防和出血控制。Rixubis是15年来获批用于B型血友病的首个新的重组凝血因子IX(rFIX),同时也是唯一一种同时适用于B型血有病患者常规预防及出血控制的rFIX。

根据美国血友病基金会(NHF)数据,B型血友病是第二种最常见类型的血友病,该病是由于血液中凝血因子IX不足所致,美国患者数为3300例,全球患者数为2.5万例。(生物谷Bioon.com)

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英文原文:FDA Approves Baxter's RIXUBIS [Coagulation Factor IX (Recombinant)] for Treatment of Children with

Hemophilia B 

Approval Based on Clinical Data Finding 39% of Pediatric Patients Less Than 12 Years of Age Experienced No Bleeds on Prophylactic Treatment

DEERFIELD, Ill., September 15, 2014 - Baxter International Inc. (NYSE:BAX) today announced that the United States Food and Drug Administration (FDA) has approved RIXUBIS [Coagulation Factor IX (Recombinant)] for routine prophylactic treatment, control and prevention of bleeding episodes, and perioperative management in children with hemophilia B. RIXUBIS was the first recombinant factor IX (rFIX) approved for routine prophylaxis and control of bleeding episodes in the U.S. for adults living with this chronic condition.

"In addition to the positive reception we've received from adult RIXUBIS patients, the approval for pediatric patients offers a valuable new option, particularly as our clinical data demonstrated a significant reduction in bleeding episodes for patients who were treated prophylactically, an important factor for this young patient population," said John Orloff, M.D., vice president of global research and development at Baxter BioScience.

The approval is based on the results of a clinical trial investigating the efficacy and safety of RIXUBIS among 23 previously-treated male patients less than

12 years of age with severe or moderately severe hemophilia B. The patients were treated with a twice-weekly RIXUBIS prophylaxis regimen (mean dose

56 IU/kg) for a mean treatment duration of six months and a mean of 54 exposure days (EDs). The median annualized bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds). Nine patients in the study (39.1%) experienced no bleeds and 23 bleeding episodes (88.5%) were treated with 1-2 infusions. There were no reports of inhibitor development, no severe allergic reactions, and no thrombotic or treatment-related adverse events among the study participants. Common adverse reactions observed in >1% of subjects in clinical studies were dysgeusia, pain in extremity, and positive test for furin antibody. These data were presented during the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA.

Baxter's application for marketing approval for RIXUBIS for adults and pediatric patients is currently under review in the European Union, with a regulatory decision expected later this year. The treatment also recently gained regulatory approval in Australia.

About RIXUBIS

RIXUBIS [Coagulation Factor IX (Recombinant)] is an antihemophilic factor indicated in adults and children with hemophilia B for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis. RIXUBIS is not indicated for induction of immune tolerance in patients with hemophilia B.

RIXUBIS was the first rFIX approved by the FDA for both routine prophylaxis and control of bleeding episodes in adults with this chronic condition. 

Important Risk Information for RIXUBIS

CONTRAINDICATIONS

RIXUBIS is contraindicated in patients who have:

Known hypersensitivity to RIXUBIS or its excipients including hamster protein

Disseminated Intravascular Coagulation (DIC)

Signs of fibrinolysis

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