诺华制药表示，美国FDA已批准其Afinitor DISPERZ（依维莫司口服混悬液片）用于2岁及以上儿童及成年结节性硬化症（TSC）相关的癫痫部分发作的辅助治疗。Afinitor Disperz是美国第一种专门用于治疗这种疾病的药物疗法。

TSC是一种罕见的遗传病，影响全球多达100万人。约85%的TSC患者受癫痫影响，与TSC相关的失控性癫痫发作可使患者衰弱。超过60%经历癫痫发作的TSC患者会停止对现有的抗癫痫疗法作出缓解反应。EXIST-3是首个证明Afinitor Disperz用于辅助治疗TSC相关癫痫部分性发作具有显着获益的临床3期试验。此外，Afinitor是唯一经批准的用于非手术治疗TSC相关的非癌性脑肿瘤(室管膜下巨细胞星形细胞瘤，或SEGA)和TSC相关的肾脏肿瘤(肾血管平滑肌脂肪瘤)的药物。

诺华肿瘤学美国公司执行副总裁Ameet Mallik表示：“Afinitor DISPERZ获得FDA批准将会对伴有癫痫部分性发作的TSC患者产生重大意义的影响。”

FDA批准Afinitor Disperz是依据一项关键临床3期有效性和安全性试验EXIST-3（EXamining everolimus In a Study of TSC）的数据。研究发现，与安慰剂相比，Afinitor Disperz在辅助治疗时，显着降低了与TSC相关的抗治疗性癫痫发作的频率。与安慰剂组(14.9%，95%CI 0.1，21.7)相比，随机服用Afinitor Disperz低剂量(LE；29.3%，95%CI 18.8，41.9；p=0.003)和高剂量(He；39.6%，95%CI 35.0，48.7；p

Afinitor通过抑制哺乳动物雷帕霉素靶蛋白(mTOR)发挥作用，mTOR是一种调节多种细胞功能的蛋白质。在TSC中，TSC 1或TSC 2基因的失活突变会导致全身错构瘤的形成，以及癫痫发作和癫痫的形成。mTOR过度激活导致神经元发育不良、轴突发育异常和树突形成、兴奋性突触电流增加、髓鞘减少和皮质层结构破坏，导致神经元发育和功能异常。在脑mTOR失调动物模型中使用mTOR抑制剂可延长其存活时间，抑制癫痫发作，防止新发性癫痫发生，并可以防止过早死亡。

Generic Name: Everolimus Tablets For Oral Suspension (e ver OH li mus)
Brand Name: Afinitor Disperz

Medically reviewed on July 4, 2018

· It is used to treat cancer.

· It is used to treat certain kinds of kidney cysts.

· It is used to help control certain kinds of seizures.

· It may be given to you for other reasons. Talk with the doctor.

· If you have an allergy to everolimus, temsirolimus, sirolimus, or any other part of Afinitor Disperz (everolimus tablets for oral suspension).

· If you are allergic to any drugs like this one, any other drugs, foods, or other substances. Tell your doctor about the allergy and what signs you had, like rash; hives; itching; shortness of breath; wheezing; cough; swelling of face, lips, tongue, or throat; or any other signs.

· If you have an infection.

· If you take any drugs (prescription or OTC, natural products, vitamins) that must not be taken with Afinitor Disperz (everolimus tablets for oral suspension), like certain drugs that are used for HIV, infections, or depression. There are many drugs that must not be taken with Afinitor Disperz (everolimus tablets for oral suspension). Your doctor or pharmacist can tell you if you are taking a drug that must not be taken with Afinitor Disperz (everolimus tablets for oral suspension).

· If you are taking another drug that has the same drug in it.

· If you are taking St. John's wort. Do not take St. John's wort with Afinitor Disperz (everolimus tablets for oral suspension). This medicine may not work as well.

· If you are breast-feeding. Do not breast-feed while you take Afinitor Disperz (everolimus tablets for oral suspension) and for 2 weeks after your last dose.

This is not a list of all drugs or health problems that interact with Afinitor Disperz (everolimus tablets for oral suspension).

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take Afinitor Disperz (everolimus tablets for oral suspension) with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

· Tell all of your health care providers that you take Afinitor Disperz (everolimus tablets for oral suspension). This includes your doctors, nurses, pharmacists, and dentists.

· You may have more of a chance of getting an infection. Wash hands often. Stay away from people with infections, colds, or flu. Some infections have been very bad and even deadly.

· High blood sugar has happened with Afinitor Disperz (everolimus tablets for oral suspension). This includes diabetes that is new or worse. Talk with the doctor.

· Tell your doctor if you have signs of high blood sugar like confusion, feeling sleepy, more thirst, more hungry, passing urine more often, flushing, fast breathing, or breath that smells like fruit.

· This medicine may cause high cholesterol and triglyceride levels. Talk with the doctor.

· Avoid grapefruit and grapefruit juice.

· This medicine may affect how wounds heal. Sometimes, people with wound healing problems have needed surgery. Call your doctor right away if you have a wound that is red, warm, painful, or swollen. Call your doctor right away if your wound opens up or if there is blood, fluid, or pus in a wound.

· Very bad and sometimes deadly lung problems have happened with Afinitor Disperz (everolimus tablets for oral suspension). Call your doctor right away if you have lung or breathing problems like trouble breathing, shortness of breath, or a cough that is new or worse.

· If you are 65 or older, use Afinitor Disperz (everolimus tablets for oral suspension) with care. You could have more side effects.

· This medicine may affect fertility. Fertility problems may lead to not being able to get pregnant or father a child. Talk with the doctor.

· If you are a man and have sex with a female who could get pregnant, you may need to protect her from pregnancy during care and for some time after care ends. Talk with your doctor.

· If you are a man and your sex partner gets pregnant while you take Afinitor Disperz (everolimus tablets for oral suspension) or within several months after your last dose, call your doctor right away.

· This medicine may cause harm to the unborn baby if you take it while you are pregnant.

· Use birth control that you can trust during care and for 2 months after care ends.

· If you get pregnant while taking Afinitor Disperz (everolimus tablets for oral suspension) or within 2 months after your last dose, call your doctor right away.

· Avoid being near anyone who has had a recent live vaccine. There are many types of live vaccines. Ask your doctor or pharmacist if you are not sure.

Use Afinitor Disperz (everolimus tablets for oral suspension) as ordered by your doctor. Read all information given to you. Follow all instructions closely.

· Take with or without food but take the same way each time. Always take with food or always take on an empty stomach.

· Take as a liquid only. Do not swallow tablets whole.

· Wear gloves when touching Afinitor Disperz (everolimus tablets for oral suspension).

· Mix with water as you have been told before drinking.

· Drink right after mixing. Throw away any part not used after 1 hour.

· If possible, a person who is pregnant or plans to get pregnant must not mix Afinitor Disperz (everolimus tablets for oral suspension). If you have questions, talk with the doctor.

· To gain the most benefit, do not miss doses.

· Keep taking Afinitor Disperz (everolimus tablets for oral suspension) as you have been told by your doctor or other health care provider, even if you feel well.

· Take Afinitor Disperz (everolimus tablets for oral suspension) at the same time of day.

· Do not switch between different forms of Afinitor Disperz (everolimus tablets for oral suspension) without first talking with the doctor.

· Talk with your doctor before getting any vaccines. Use with Afinitor Disperz (everolimus tablets for oral suspension) may either raise the chance of an infection or make the vaccine not work as well.

· To prevent mouth irritation or mouth sores, follow what your doctor has told you to do. If you have mouth irritation or mouth sores, do not use mouth rinses that have alcohol, peroxide, iodine, or thyme in them. If you have questions, talk with your doctor.

· Have blood work checked as you have been told by the doctor. Talk with the doctor.

· Check your blood sugar as you have been told by your doctor.

· If you are able to get pregnant, a pregnancy test will be done to show that you are NOT pregnant before starting Afinitor Disperz (everolimus tablets for oral suspension). Talk with your doctor.

What do I do if I miss a dose?

· Take a missed dose as soon as you think about it.

· If it has been 6 hours or more since the missed dose, skip the missed dose and go back to your normal time.

· Do not take 2 doses at the same time or extra doses.

See also: Dosage Information (in more detail)

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

· Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.

· Signs of infection like fever, chills, very bad sore throat, ear or sinus pain, cough, more sputum or change in color of sputum, pain with passing urine, mouth sores, or wound that will not heal.

· Signs of liver problems like dark urine, feeling tired, not hungry, upset stomach or stomach pain, light-colored stools, throwing up, or yellow skin or eyes.

· Redness or irritation of the palms of hands or soles of feet.

· A burning, numbness, or tingling feeling that is not normal.

· Flushing.

· Chest pain.

· Any unexplained bruising or bleeding.

· A fast heartbeat.

· Dizziness or passing out.

· A heartbeat that does not feel normal.

· Seizures.

· Muscle pain or weakness.

· Period (menstrual) changes. These may include a missed period.

· Mood changes.

· Change in the way you act.

· Swelling.

· Very bad and sometimes deadly kidney problems have happened with Afinitor Disperz (everolimus tablets for oral suspension). Call your doctor right away if you are unable to pass urine or if you have blood in the urine or a change in the amount of urine passed.

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

· Pimples (acne).

· Feeling tired or weak.

· Headache.

· Not able to sleep.

· Upset stomach or throwing up.

· Loose stools (diarrhea).

· Hard stools (constipation).

· Mouth irritation or mouth sores.

· Change in taste.

· Not hungry.

· Weight loss.

· Dry mouth.

· Dry skin.

· Change in nails.

· Hair loss.

· Joint pain.

· Back pain.

· Pain in arms or legs.

· Muscle spasm.

· Nosebleed.

· Belly pain.

· Nose or throat irritation.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-FDA-1088. You may also report side effects at http://www.fda.gov/medwatch.

See also: Side effects (in more detail)

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

· Store in the original container at room temperature.

· Protect from light.

· Store in a dry place. Do not store in a bathroom.

· Keep all drugs in a safe place. Keep all drugs out of the reach of children and pets.

· Throw away unused or expired drugs. Do not flush down a toilet or pour down a drain unless you are told to do so. Check with your pharmacist if you have questions about the best way to throw out drugs. There may be drug take-back programs in your area.

· If your symptoms or health problems do not get better or if they become worse, call your doctor.

· Do not share your drugs with others and do not take anyone else's drugs.

· Keep a list of all your drugs (prescription, natural products, vitamins, OTC) with you. Give this list to your doctor.

· Talk with the doctor before starting any new drug, including prescription or OTC, natural products, or vitamins.

· Some drugs may have another patient information leaflet. Check with your pharmacist. If you have any questions about Afinitor Disperz (everolimus tablets for oral suspension), please talk with your doctor, nurse, pharmacist, or other health care provider.

· If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.