英文药名：FABRAZYME(AGALSIDASE BETA INTRAVENOUS) 中文药名：重组人α-半乳糖苷酶A注射液 生产厂家：Genzyme Corporation 药品介绍 Fabrazyme(重组人α-半乳糖苷酶A)冻晶注射剂首个治疗布里雷（Fabry）病，防止神经性疼痛、慢性肾功能不全、心脏病变等发生的新药。 Fabrazyme推荐治疗 剂量： 16岁以上青少年及成人每两星期接受一次静脉输注液，剂量为1mg/kg。起初20个星期，药品输注速率为0.25mg/min。输注时间不可少于2小时。 相互作用： 不可与Chloroquine、Aminodatone、Benoquin、Gentamicin一起使用。 注意事项： 对此药过敏、中等至严重的高血压(于输注时可能恶化)、肾衰竭(对于血浆肌酸酐大于2.5mg/dL患者的研究不足)及发烧病患(会恶化)应避免使用。监控治疗指标为定期测量血浆中GL-3浓度，正常浓度应低于1.2ng/ml。 副作用： 发生频率大于10%的症状包括：全身性症状－寒颤、对温度变化敏感、发烧、四肢疼痛，呼吸系统－支气管痉挛、喉咙紧闷，胃肠道－恶心、呕吐，中枢及周围神经系统－头痛、震颤，心血管－四肢水肿、高血压，肌肉骨胳系统－肌痛。 包装规格 agalsidase beta (Fabrazyme®) 5 mg powder for concentrate for solution for infusion agalsidase beta (Fabrazyme®) 35 mg powder for concentrate for solution for infusion Fabrazyme®(agalsidase beta) FABRAZYME Rx Generic Name and Formulations: Agalsidase beta 5mg, 35mg; per vial; pwd for IV infusion after reconstitution; preservative-free. Company: Genzyme Corporation Indications for FABRAZYME: Fabry disease. Adults and Children: <8yrs: not recommended. ≥8yrs: Pretreat with antipyretic and antihistamine. Give by IV infusion. 1mg/kg infused every 2 weeks. Initial infusion rate should be ≤0.25mg/min; if tolerated, may increase infusion rate in increments of 0.05–0.08mg/min with each subsequent infusion. <30kg: max rate should remain at 0.25mg/min. ≥30kg: administration duration should not be less than 1.5hrs. Slow rate in the event of infusion reactions. Rechallenge (see literature): initially should be a low dose and infusion rate (eg, 0.5mg/kg at 0.01mg/min); once tolerated, may increase dose to 1mg/kg, titrate rate slowly upwards (doubled every 30 minutes up to max 0.25mg/min). Pharmacological Class: Recombinant human alpha-galactosidase A enzyme. Warnings/Precautions: Have appropriate medical support measures available if anaphylaxis or severe allergic reactions develop. Compromised cardiac function; monitor closely. Consider testing for IgE antibodies in patients who experienced suspected allergic reactions; if positive, may be rechallenged. Pregnancy (Cat.B). Nursing mothers. Adverse Reactions: Infusion reactions (chills, fever, feeling hot or cold, dyspnea, nausea, flushing, headache, vomiting, paresthesia, fatigue, pruritus, pain in extremity, hypertension, chest pain, throat tightness, abdominal pain, dizziness, tachycardia, nasal congestion, diarrhea, peripheral edema, myalgia, back pain, pallor, bradycardia, urticaria, hypotension, face edema, rash, somnolence; if severe, discontinue immediately), anaphylaxis and allergic reactions (may be fatal; discontinue immediately). Note: Register pregnant patients at www.fabryregistry.com or call (800) 745-4447. How Supplied: Single-use vial—1 Fabrazyme is the first specific treatment for Fabry disease, an inherited and often life-threatening disorder characterized by the progressive build-up of a substance known as GL-3 within the cells. As an enzyme replacement therapy (ERT), Fabrazyme replaces the enzyme responsible for lowering GL-3 that is missing or deficient in people with Fabry disease. Sanofi Genzyme is committed to providing information, resources, and support to address the unique challenges of living with Fabry disease. This site has been developed to help answer questions you may have if you or someone you know has been diagnosed with Fabry disease or is starting treatment with Fabrazyme. Indication and Usage Fabrazyme® (agalsidase beta) is used to treat patients with Fabry disease. Fabrazyme lowers the amount of a substance called globotriaosylceramide (GL-3), which builds up in cells lining the blood vessels of the kidney and certain other cells. The lowering of GL-3 suggests that Fabrazyme may improve how Fabry disease affects your body; however a relationship of lower GL-3 to specific signs and symptoms of Fabry disease has not been proven. Important Safety Information Life-threatening severe allergic (anaphylactic) reactions have been seen in patients during Fabrazyme infusions. Approximately 1% of patients who have received Fabrazyme either during a clinical study or after Fabrazyme was approved have experienced anaphylactic or severe allergic reactions during their infusion. These reactions have included: localized swelling of the face, mouth and throat, narrowing of breathing airways, low blood pressure, hives, difficulty swallowing, rash, trouble breathing, flushing, chest discomfort, itching and nasal congestion. People who have experienced these reactions have required treatment including heart/lung resuscitation, oxygen, fluids given through the vein, hospitalization, and have needed treatment with inhaled drugs called beta-adrenergic agonists to help open the breathing airways, antihistamines, epinephrine (also known as adrenalin), and a medication given through the vein called a corticosteroid (or steroid) which helps to decrease the body’s allergic reaction by decreasing inflammation. If you experience a severe allergic or anaphylactic reaction, your healthcare professional will immediately stop the infusion of Fabrazyme and provide you the necessary emergency medical treatment. Because of the possibility that severe allergic reactions may occur, appropriate medical support should be available during your Fabrazyme infusion. •For patients who have had reactions to their infusions, it is recommended that they be given anti-fever and antihistamine medications right before their next infusions. •Infusion reactions have happened in some patients even after taking these medications and steroids by mouth before their infusions. •If an infusion reaction occurs, slowing the infusion rate, stopping the infusion for a short time and/or giving more anti-fever and antihistamine medications and or steroids may improve the symptoms. •If severe infusion reactions happen, your healthcare professional should consider stopping the Fabrazyme infusion right away and should provide medical care for your condition. •Severe reactions are generally managed by giving antihistamine medications, corticosteroids, fluids through the vein, and/or oxygen when needed. •Because severe infusions reactions may happen, medical treatment should be readily available during your Fabrazyme infusion. Providing Fabrazyme to patients who have experienced severe or serious allergic reactions to Fabrazyme should only be done after carefully considering the risks and benefits of continuing the treatment, and only under the direct supervision of a qualified healthcare professional and with appropriate medical support readily available. The most common side effects reported with Fabrazyme are infusion reactions, some of which were severe. When Fabrazyme was tested in clinical studies, infusion reactions occurred in approximately 50-55% of patients. Serious and/or frequently occurring side effects (occurring in 5% or more of the patients) thought to be related to Fabrazyme have included one or more of the following: chills, fever, feeling hot or cold, trouble breathing, nausea, flushing of the skin, headache, vomiting, burning and/or tingling sensation, fatigue, itching, pain in the hands and feet, high blood pressure, chest pain, throat tightness, abdominal pain, dizziness, rapid heart rate, nasal congestion, diarrhea, swelling in the legs, muscle pain, back pain, paleness of the skin, slow heart rate, hives, low blood pressure, face swelling, rash and sleepiness. People with advanced Fabry disease may have heart problems, which may put them at a higher risk for severe complications from infusion reactions, and these patients should be watched closely during their infusion if the decision is made to give them Fabrazyme. Other serious side effects that were seen in the clinical studies included stroke, pain, lack of muscle coordination, slow or irregular heartbeat, stopping of the heartbeat, decreased blood pumped by the heart, dizziness, hearing loss, and kidney problems resulting in too much protein leaving the body in the urine (nephrotic syndrome). These side effects also occur as part of Fabry disease. Severe and serious infusion reactions have been reported since Fabrazyme has been approved, some of which were life threatening including anaphylactic shock (a severe allergic reaction). In addition to the above side effects, the following have been reported since Fabrazyme has been approved: joint pain, lack of strength or energy, redness of the skin, increased sweating, reactions at the place where the catheter to give the infusion is placed, increased tearing from the eyes, allergic inflammation of blood vessels, enlarged lymph nodes, decreased sensitivity to touch or pressure, decreased sensitivity of the mouth, sensations of an abnormal heartbeat, runny nose, low oxygen (in general), and low oxygen levels reaching different parts of the body. Since Fabrazyme has been approved, there have been side effects that resulted in death that may or may not be related to the use of Fabrazyme. These included: the heart and/or lungs stop working (known as cardiorespiratory arrest, respiratory failure, and/or cardiac failure), life-threatening infection in the blood stream (known as sepsis), stroke, heart attack, kidney failure, and pneumonia. Some of these side effects were reported in Fabry disease patients with significant underlying disease. The safety and effectiveness of Fabrazyme in patients younger than 8 years of age have not been studied. Most patients taking Fabrazyme who develop IgG antibodies, which are commonly produced by your immune system in response to things it does not recognize as naturally being part of your body, do so within the first three months of taking the medication. In children, the development of these IgG antibodies was associated with Fabrazyme staying in the body for a longer time (prolonged half-life), which was rarely seen in adult patients. In the clinical studies, a few patients developed IgE antibodies or a reaction to an allergy skin test specific to Fabrazyme. IgE antibodies are usually produced by the body’s immune system during an allergic reaction. Your doctor should consider testing for IgE antibodies if you experience suspected allergic reactions and consider the risks and benefits of continued treatment with Fabrazyme if you have IgE antibodies against Fabrazyme.