DEFITELIO(DEFIBROTIDE SODIUM)SOLUTION IV获FDA批准在接受来自血或骨髓干细胞移植患者对罕见病第一个治疗
FDA的药品评价和研究中心中血液学和肿瘤室主任Richard Pazdur，M.D.说：“准满足移植社会接受化疗和HSCT患者治疗这个罕见但频繁致命性合并症显著需求。” 优先审评状态和孤儿药物指定
批准日期：2016年3月30日；公司 Jazz Pharmaceuticals plc
DEFITELIO(去纤维钠)注射液是在一个单次-患者-使用，透明玻璃小瓶作为一个清澈，浅黄色至棕色，无菌，无防腐剂溶液为静脉输注供应。每小瓶(NDC 68727-800-01)含200 mg/2.5 mL(在一个浓度80 mg/mL)的去纤维钠。
贮存DEFITELIO(去纤维钠)注射液在20°C-25°C(68°F-77°F)；外出允许15° C至30°C(59°F至 86°F)间(见USP控制室温)。
First and Only FDA-Approved Therapy for Patients with this Rare, Potentially Fatal Complication
FDAgranted marketing approval for Defitelio® (defibrotide sodium) for the treatment of adult and pediatric patients with hepatic VOD, also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following HSCT.
"FDA's approval of Defitelio underscores the importance of Defitelio to children and adults as the first and only proven treatment for this rare and often deadly complication of stem-cell transplantation. Defitelio is a clinically significant therapeutic advance because it is a potentially curative intervention for patients with VOD, which may save lives with a single course of therapy. Before today, patients in the U.S. had no approved options," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "The commercial availability of Defitelio in the U.S. demonstrates Jazz Pharmaceuticals' continued commitment to bringing meaningful new treatments to patients to fill high unmet medical needs."
The FDA approval of Defitelio is supported by efficacy data from three clinical studies in patients with hepatic VOD with renal or pulmonary dysfunction following HSCT who were treated with Defitelio at the recommended 6.25 mg/kg every 6 hours; results are provided in the table below.
The safety of Defitelio to support approval is based on data from 176 patients in the clinical development program for the treatment of VOD with renal and/or pulmonary dysfunction following HSCT who were treated with Defitelio. The most common adverse reactions (incidence =10% and independent of causality) with Defitelio treatment were hypotension (low blood pressure), diarrhea, vomiting, nausea and epistaxis (nose bleeds).The most common serious adverse reactions (incidence =5% and independent of causality) were hypotension (11%) and pulmonary alveolar hemorrhage (7%).
"VOD/SOS is a devastating condition, which can develop without warning after stem-cell transplantation and can progress rapidly causing severe kidney or lung dysfunction and lead to multi-organ failure. Thus, it can derail a patient's recovery from the curative intent of a stem-cell transplant, with patients who develop VOD/SOS and multi-organ failure facing an overall mortality rate of over 80%," said Paul G. Richardson, M.D., director of clinical research at the LeBow Institute for Myeloma Therapeutics and the Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute and the RJ Corman Professor of Medicine at Harvard Medical School. "Defitelio thus fulfills an unmet need having shown a consistent Day+100 patient survival benefit across three large prospective studies. Importantly, Defitelio provides transplant teams with the first approved treatment option that can help return patients to the road to recovery."
Defitelio® (defibrotide sodium) injection 80mg/mL is indicated for the treatment of adult and pediatric patients with hepatic VOD, also known as SOS, with renal or pulmonary dysfunction following HSCT. The FDA granted the Defitelio application priority review status. Defitelio also received orphan drug designation for the treatment of hepatic VOD.
Important Safety Information
Defitelio should not be given to patients who are:
•Currently taking anticoagulants or fibrinolytics
•Allergic to Defitelio or any of its ingredients
Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.
Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.
The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds. Please see full Prescribing Information for Defitelio before prescribing.
In Europe, defibrotide is marketed under the name Defitelio®?(defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients undergoing HSCT therapy. It is the first and only approved treatment in Europe for severe VOD. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy.
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Miscellaneous gastrohepatic disorders Only 4 drugs may be compared at once
Generic Name and Formulations:
Defibrotide sodium 200mg/2.5mL; soln for IV infusion after dilution; preservative-free.
Jazz Pharmaceuticals plc
Select therapeutic use: Miscellaneous gastrohepatic disorders
RECENT UPDATES Monograph added.
Indications for DEFITELIO:
Treatment of hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation.
Adults and Children:
Confirm patient is hemodynamically stable on ≤1 vasopressor. Give by IV infusion over 2hrs. 6.25mg/kg every 6hrs for at least 21 days; if VOD unresolved after 21 days, continue until resolution or up to max 60 days. Treatment modification: see full labeling.
Concomitant systemic anticoagulant or fibrinolytic therapy.
Increased risk of bleeding; monitor. Active bleeding: do not initiate. Discontinue permanently if severe or life-threatening hypersensitivity reaction or recurrent significant bleeding occurs. Withhold if persistent, severe or potentially life-threatening bleeding occurs; consider resuming when bleeding has stopped. Discontinue ≥2hrs prior to an invasive procedure; resume as soon as any procedure-related bleeding risk has resolved. Pregnancy: potential risk of miscarriage. Nursing mothers: not recommended.
See Contraindications. Discontinue concomitant anticoagulants and fibrinolytics before initiating Defitelio.
Hypotension, diarrhea, vomiting, nausea, epistaxis; hemorrhage, hypersensitivity reactions.
Defitelio injection 200mg/2.5mL(80mg/mL)/vial 10vial/box
Defitelio注射液 200毫克/2.5毫升（80毫克/毫升）/瓶 10瓶/盒
Jazz Pharmaceuticals, Inc