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首页 » FDA信息 » 接班重磅药Exjade 诺华慢性铁过载口服新药Jadenu获FDA批准

接班重磅药Exjade 诺华慢性铁过载口服新药Jadenu获FDA批准

来源:生物谷 2015-04-02 09:30

2015年4月2日讯 /生物谷BIOON/ --瑞士制药巨头诺华(Novartis)近日宣布,FDA已批准新药Jadenu(deferasirox,地拉罗司):(1)用于2岁及以上患者因输血治疗所致的慢性铁过载(CIO)的治疗;(2)用于10岁及以上非输血依赖型地中海贫血综合征(NTDT)患者慢性铁过载(CIO)的治疗。

Jadenu被开发作为诺华已上市药物Exjade(deferasirox)的接班人,该药在美国已上市10年,是处方量最大的一种铁螯合剂,年销售额近10亿美元。

Jadenu是Exjade的一种新口服配方,是首个可直接吞服的每日一次口服铁螯合剂。Exjade则是一种分散片,必须在液体中混合并在饭前至少30分钟空腹服用。Jadenu则在有无便餐的情况下均可直接吞服,从而大大简化了慢性铁过载(CIO)的治疗管理。Jadenu的上市,对于需要定期服用铁螯合剂治疗以清除体内过量铁的广大患者而言,是一个大好消息。

Jadenu的加速批准,是基于该药在临床试验中降低肝脏铁浓度和血清铁蛋白水平的疗效数据。目前,诺华正在开展临床研究进一步调查Jadenu的长期疗效和安全性。当前,许多镰状细胞病、地中海贫血或骨髓增生异常综合症患者需要频繁输血治疗,相应地也需要长期的日常铁螯合治疗。

慢性铁过载(CIO)是由于过量的铁长期在机体组织和器官(特别是心脏和肝脏)中积累所致,多见于频繁输血治疗的贫血患者,若不及时治疗可导致致命性的肝脏和心脏损伤。此外,慢性铁过载(CIO)也可能发展成一种名为血色素沉着症(hemochromatosis)的疾病,该病特征为虚弱、疲劳、关节或腹部疼痛甚至器官衰竭。因此,有效去除体内过量的铁,对于频繁接受输血治疗的患者而言极为重要。deferasirox(地拉罗司)是一种铁鳌合剂,用于改善铁过载疗效显著。

不过,需要注意的是,Jadenu也有相关的肾脏疾病风险,该药禁用于伴有严重肾脏或肝脏问题、晚期癌症、高危骨髓增生异常综合征(MDS)、过敏、低血小板计数的患者。除美国外,诺华也已向其他主要市场提交了Jadenu的监管申请。(生物谷Bioon.com)

英文原文:Novartis announces FDA approval for JadenuTM to simplify treatment administration for patients with chronic iron overload

Jadenu (deferasirox), a new formulation of Exjade (deferasirox), is the only once-daily oral tablet for iron chelation
Jadenu, taken with or without food, simplifies daily treatment administration for patients with chronic iron overload
Chronic iron overload is a serious condition that can affect people with sickle cell disease, thalassemia and myelodysplastic syndromes
Basel, March 30, 2015 - Novartis announced today that the US Food and Drug Administration (FDA) has approved JadenuTM (deferasirox) tablets, a new oral formulation of Exjade® (deferasirox) tablets for oral suspension, for the treatment of chronic iron overload due to blood transfusions in patients 2 years of age and older, and chronic iron overload in non-transfusion-dependent thalassemia syndromes (NTDT) in patients 10 years of age and older. Jadenu is the only once-daily oral iron chelator that can be swallowed whole.

Many patients with sickle cell disease, thalassemia or myelodysplastic syndromes need repeated blood transfusions and consequently, long-term daily chelation therapy. Jadenu oral tablets can be taken in a single step, with or without a light meal, simplifying administration of treatment for chronic iron overload. Exjade is a dispersible tablet that must be mixed in liquid and taken on an empty stomach. Jadenu is approved under accelerated approval based on a reduction of liver iron concentrations and serum ferritin levels. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

"Novartis has had a long-term commitment to improving the lives of patients with chronic iron overload," said Bruno Strigini, President, Novartis Oncology. "Exjade transformed iron chelation therapy. We responded to feedback from patients and their physicians, and now Jadenu, by simplifying treatment administration, offers an important new option to help meet these patients' needs."

Chronic iron overload is a life-threatening cumulative toxicity that results from blood transfusions required to treat sickle cell disease, myelodysplastic syndromes, thalassemia and other conditions. Chronic iron overload also can occur in patients with NTDT due to increased iron absorption in the stomach and intestines[1],[2]. If left untreated, chronic iron overload can damage the liver and heart[3],[4].

Jadenu contains deferasirox, the same active ingredient that is in Exjade, a medicine that has been used by patients with chronic iron overload for almost 10 years. Exjade currently is the most-prescribed chelator in the United States[5].

"Jadenu is an exciting development for patients with chronic iron overload who have been eager for alternative treatment options," said Dr. Elliott Vichinsky, Director of Hematology and Oncology at the University of California, San Francisco (UCSF) Benioff Children's Hospital Oakland and Professor, UCSF School of Medicine. "Taking iron chelation therapy every day has sometimes been a challenge for them. The administration of Jadenu oral tablets once a day is simple."

Novartis has submitted additional regulatory applications for Jadenu in other countries worldwide.

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