美国FDA批准庞培氏病（Pompe）的首个药物 Myozyme
Myozyme（通用名：alglucosidase alfa, rhGAA）是首个治疗庞培氏病（Pompe/糖原累积症Ⅱ型）的药物。
美国食品暨药物管理局（FDA）批准了Myozyme的生物制品许可申请(biologics license application，BLA)，Myozyme是首个治疗庞培氏病（Pompe）的药物。
Myozyme已被批准为FDA指定罕见病药，并被批准优先审查。罕见病产品的开发，用于治疗在美国影响少于20万人的罕见疾病或症状，《罕见病药品法》（Orphan Drug Act）给获得一个指定罕见病药上市批准的首个申请者提供了7年的市场独占期。
FDA批准Myozyme通过静脉输液给药。在两项独立临床试验中，对39位1个月至3.5岁的患有庞培氏病的婴幼儿的首次给药评估了Myozyme的安全性及有效性。
使用该药治疗的婴幼儿患者与未使用该药的同龄同病况患者的已知高致命性相比，无需侵入型吸氧（invasive ventilatory）维持生命的存活率比预计的明显增高。该药在其他形式庞培氏病中的安全性和有效性，尚未得到充分研究。
对Myozyme最严重的不良反应报道是心脏与肺衰竭及过敏性休克。最常见的反应包括肺炎、呼吸衰竭与呼吸困难、感染及发烧。Myozyme标签中包含的一个加框警告称可能存在威胁生命的过敏反应。
Myozyme由美国马塞诸塞州剑桥市的Genzyme公司生产

•英文品名：Myozyme
•成份名：Alglucosidase Alfa
•劑型：注射劑
•劑量單位：50mg/vial
•規格包裝：1 vial/box
•適應症/用途：龐貝氏症

Myozyme
Generic Name: alglucosidase alfa (injection) (AL gloo KOE si dase AL fa)
Brand Names: Myozyme
What is Myozyme?
Myozyme (alglucosidase alfa) contains an enzyme that naturally occurs in the body in healthy people. Some people lack this enzyme because of a genetic disorder. Myozyme helps replace this missing enzyme in such people.
Myozyme is used to treat a glycogen storage disorder called Pompe disease, (also called GAA deficiency).
Myozyme may also be used for purposes other than those listed in this medication guide.
Important information about MyozymeBefore receiving Myozyme, tell your doctor if you are allergic to mice, hamsters, or drug products made with "murine" proteins.
Myozyme is given as an injection through a needle placed into a vein. You will receive this injection with an infusion pump in a clinic or hospital setting. The medicine must be given slowly through an IV infusion, and can take up to 4 hours to complete.
Myozyme is usually given every 2 weeks.
Some people receiving an injection of Myozyme have had a reaction to the infusion (when the medicine is injected into the vein). Tell your caregiver right away if you feel dizzy, nauseated, light-headed, sweaty, itchy, or have a fast heartbeat, cold hands, blue lips, tightness in your chest or throat, wheezing, or trouble breathing during the injection.
Before receiving MyozymeYou should not use Myozyme if you are allergic to alglucosidase alfa.
If you have any of these other conditions, you may need a dose adjustment or special tests to safely use this medication:
heart disease;
lung disease or breathing disorder;
if you are allergic to mice, hamsters, or drug products made with "murine" proteins.
FDA pregnancy category B. Myozyme is not expected to be harmful to an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant during treatment. It is not known whether alglucosidase alfa passes into breast milk or if it could harm a nursing baby. Do not use this medication without telling your doctor if you are breast-feeding a baby.
How is Myozyme given?
Myozyme is given as an injection through a needle placed into a vein. You will receive this injection with an infusion pump in a clinic or hospital setting. The medicine must be given slowly through an IV infusion, and can take up to 4 hours to complete.
Myozyme is usually given every 2 weeks.
Before each injection, tell your doctor if you have recently been sick with a cold, flu, or other illness.
Your name may need to be listed on a patient registry for people with Pompe disease when you start using this medication.
What happens if I miss a dose?Contact your doctor if you miss a dose of this medication.
What happens if I overdose?Seek emergency medical attention if you think you have received too much of this medicine. An overdose of Myozyme is not expected to produce life-threatening symptoms.
What should I avoid while receiving Myozyme?There are no restrictions on food, beverages, or activity while receiving Myozyme unless your doctor has told you otherwise.
Myozyme side effectsSome people receiving an injection of Myozyme have had a reaction to the infusion (when the medicine is injected into the vein). Tell your caregiver right away if you feel dizzy, nauseated, light-headed, sweaty, itchy, or have a fast heartbeat, cold hands, blue lips, tightness in your chest or throat, wheezing, or trouble breathing during the injection.
Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficulty breathing; swelling of your face, lips, tongue, or throat. Call your doctor at once if you have any of these serious side effects:
fast, slow, or uneven heartbeats;
weak pulse, fainting, slow breathing (breathing may stop);
chest pain or heavy feeling, pain spreading to the arm or shoulder, sweating, general ill feeling;
ongoing cough with heaviness in your chest;
tremors or shaking;
feeling light-headed, fainting;
muscle weakness; or
skin ulcers.
Other less serious Myozyme side effects are more likely to occur, such as:
headache;
fever;
diarrhea, nausea, vomiting, stomach pain;
pain or fullness in your ear;
runny or stuffy nose, sore throat, cough; or
feeling irritable or agitated.
What other drugs will affect Myozyme?There may be other drugs that can affect Myozyme. Tell your doctor about all the prescription and over-the-counter medications you use. This includes vitamins, minerals, herbal products, and drugs prescribed by other doctors. Do not start using a new medication without telling your doctor.
Where can I get more information?Your pharmacist has more information about Myozyme written for health professionals that you may read.
Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.
Every effort has been made to ensure that the information provided by Cerner Multum, Inc. ('Multum') is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Multum information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Multum does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Multum's drug information does not endorse drugs, diagnose patients or recommend therapy. Multum's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners. The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Multum does not assume any responsibility for any aspect of healthcare administered with the aid of information Multum provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are receiving, check with your doctor, nurse or pharmacist.
价格：MYOZYME 50MG VIAL D/SHIP 50ML
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产地：美国
原产地英文商品名:
MYOZYME 50MG VIAL
原产地英文药品名:
ALGLUCOSIDASE ALFA
中文参考商品译名:
MYOZYME 50毫克瓶
生产厂家中文参考译名:
Genzyme公司
生产厂家英文名:
Genzyme