LIVING PROOF. 23% Reduction in the risk of death compared with standardof-care. A guide to combination treatment with RYDAPT

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1 My LIVING PROOF THAT TREATING NEWLY DIAGNOSED FLT3-POSITIVE AML HAS CHANGED 23% Reduction in the risk of death compared with standardof-care chemotherapy alone A specific median overall survival (the time point at which half the patients were still alive) could not be reliably estimated A guide to combination treatment with RYDAPT INDICATION for RYDAPT (midostaurin) capsules RYDAPT is an oral prescription medicine used in combination with certain chemotherapy medicines to treat adults with newly diagnosed acute myeloid leukemia (AML) who have a defect in a gene called FLT3. Your doctor will perform a test to make sure RYDAPT is right for you. RYDAPT should not be used alone to induce remission in people with AML. It is unknown if RYDAPT is safe and effective in children. IMPORTANT SAFETY INFORMATION for RYDAPT Allergic Reactions Do not take RYDAPT if you are allergic to midostaurin or any of the ingredients in RYDAPT If you develop signs of an allergic reaction, seek medical help immediately Signs of an allergic reaction include trouble breathing, flushing, chest pain, throat tightness, and swelling of the face, lips, tongue, or throat People depicted throughout this brochure are not actual patients.

2 UNDERSTANDING AML Being diagnosed with AML can be an overwhelming experience. You may feel like your whole life has been turned upside down. At Novartis, we understand that you re facing new challenges, but we want you to know that we re committed to providing you with support. Understanding AML and how it s managed is an important first step. The information in this brochure is meant to help you and those close to you learn more about AML and what you might expect throughout your journey. You ll also learn about your treatment with RYDAPT (midostaurin) capsules and how it may play an essential role in the overall plan that your health care team has created for you. What is AML? AML is a type of blood cancer that primarily affects your white blood cells. In AML, the myeloid stem cells usually become a type of immature white blood cell called myeloblasts (or myeloid blasts). The myeloblasts in AML are abnormal and do not become healthy white blood cells. Leukemia cells often reproduce quickly, but in most cases the problem is that they don t die when they should. Acute leukemias, like AML, are fast growing because normal blast cells divide quickly. The leukemia cells don t divide any more often than normal blast cells do. However, they don t stop dividing when normal blast cells would. IMPORTANT SAFETY INFORMATION (CONTINUED) Pregnancy, Breastfeeding, Fertility RYDAPT should not be used during pregnancy since it may harm an unborn baby If you become pregnant, think you may be, or are planning to be, tell your doctor right away If you are able to become pregnant, your doctor should perform a pregnancy test within 7 days before you start RYDAPT. Effective birth control should be used during treatment and for at least 4 months after the last RYDAPT dose Men taking RYDAPT who have female partners who are able to become pregnant should use effective birth control during treatment with RYDAPT and for at least 4 months after the last dose Pregnancy registry: There is a pregnancy registry that monitors the health of females and their babies exposed to RYDAPT during pregnancy. Females who have taken RYDAPT during pregnancy, or who have been exposed to RYDAPT during pregnancy through a male partner taking RYDAPT, should contact Novartis Pharmaceuticals Corporation at or at Do not breastfeed during treatment with RYDAPT and for at least 4 months after the final dose RYDAPT may impair fertility in males and females. You should discuss this with your doctor before starting treatment 2

3 DIAGNOSING AML How is AML diagnosed? Your doctor uses blood tests and a bone marrow biopsy to determine if you have AML. The table on the next page illustrates the different blood cell counts that can be found in patients with AML. Changes may be seen in the white blood cells, red blood cells, and platelets. The lab may also identify changes in blasts. AML is the most common type of acute leukemia affecting adults. In 2017, an estimated 21,380 people in the United States will be diagnosed with AML. IMPORTANT SAFETY INFORMATION (CONTINUED) Lung Problems RYDAPT may cause serious lung problems that may lead to death when used alone or in combination with other chemotherapy medicines If you develop a new or worsening cough, shortness of breath, or chest discomfort, contact your doctor immediately. These may be signs of serious lung problems A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 3

4 DIAGNOSING AML (CONTINUED) Blood-test results that may lead to a diagnosis of AML Blood cell type White blood cells Blasts circulating in the blood Red blood cells Platelets Can be low or high High white blood cell count may represent an excess of immature white blood cells Variable number Low Low Count Your health care provider may also order additional tests to identify the specific type of AML that you have. One test that your doctor is likely to use is genetic screening. AML is linked with mutations of certain types of genes. Genetic screening may identify genes that have mutated, including one of the most common AML-related mutations, FLT3. Approximately 30% of patients with AML harbor some form of FLT3 mutation. Identifying such mutations may help your health care provider determine a tailored plan for treating your AML. IMPORTANT SAFETY INFORMATION (CONTINUED) Side Effects Most common side effects reported during RYDAPT treatment for acute myeloid leukemia (AML) included: low level of white blood cells with fever (febrile neutropenia) nausea vomiting headache bruising nose bleeds device-related infection high blood sugar levels redness, pain, or ulcers inside muscle or bone pain (hyperglycemia) the mouth (mucositis) upper respiratory infection 4 If side effects including nausea, vomiting, and diarrhea occur, get worse, or do not go away during treatment with RYDAPT, you should contact your doctor immediately. Your doctor may need to decrease your dose, temporarily stop, or completely stop your treatment with RYDAPT These are not all of the possible side effects of RYDAPT. For more information, ask your doctor or pharmacist

5 WHAT IS FLT3-POSITIVE AML, AND HOW DOES IT AFFECT MY TREATMENT OPTIONS? Your doctor may have mentioned that you have a specific type of AML known as FLT3-positive AML. FLT3 is a gene that can help determine which AML treatments may be most appropriate for you, allowing your health care provider to design a tailored treatment plan. The DNA in your cells can become altered (or mutated), and when the FLT3 gene is affected, it can play a major role in your cancer s development. About 3 out Approximately 3 of out10 ofof 10patients patients with with AML AML have have the mutated FLT3 gene the mutated FLT3 gene Your FLT3 status can help you understand why RYDAPT may be a treatment option for you. It is unknown if RYDAPT is safe and effective in children. A GUIDE TO COMBINATION TREATMENT WITH RYDAPT If your doctor has found that your FLT3 gene is mutated, he or she may have told you that you are positive for FLT3. This is one of the reasons you have been prescribed RYDAPT (midostaurin) capsules, a medicine to treat this type of AML. 5

6 RYDAPT CAN BE AN EFFECTIVE OPTION IN YOUR FLT3-POSITIVE AML TREATMENT PLAN RYDAPT (midostaurin) capsules is an oral medicine approved by the FDA to treat newly diagnosed FLT3-positive AML. RYDAPT belongs to a group of therapies called protein kinase inhibitors. Protein kinases are enzymes that send signals to help cells grow and divide. Blocking them may help prevent leukemia cells from growing. RYDAPT is thought to block the action of several kinases of leukemia cells, which may help prevent these cells from dividing and growing. At the start of your newly diagnosed FLT3-positive AML treatment, RYDAPT is always used together with chemotherapy. We will look more closely at possible paths on the following page. How is AML treated? Following your initial AML diagnosis, your doctor will order a test to determine your FLT3 status. If you test positive for FLT3, you may receive at least one of the following treatments: RYDAPT plus chemotherapy Chemotherapy Stem cell transplantation RYDAPT is the first therapy for newly diagnosed FLT3-positive AML approved by the FDA in over 25 years. 6 Please see Important Safety Information throughout this brochure and the Summary of Important Information on pages 19 to 21. It is unknown if RYDAPT is safe and effective in children.

7 YOUR TREATMENT PLAN WILL MOST LIKELY INCLUDE SEVERAL STAGES INDUCTION (1 to 2 cycles*) Chemotherapy: Days 1 to 7 Because AML is a disease that can get worse quickly, it s important that you start treatment for AML right after your diagnosis. This initial stage of treatment, which is given during your stay in the hospital, is called induction therapy and will most likely include chemotherapy. The goals of induction therapy are to: Kill as many AML cells as possible Get blood counts back to normal Get rid of all signs of the disease for an extended period of time During your induction with chemotherapy as part of the diagnostic workup, you may be tested to determine your FLT3 status. CONSOLIDATION (up to 4 cycles) Chemotherapy: Days 1, 3, and 5 Because there may be AML cells remaining after the first phase of your treatment (the induction phase), you may need additional therapy. This may include additional rounds of chemotherapy, which can be given while you are in the hospital or as outpatient therapy. Your doctor will discuss what the best treatment plan for you may be. RYDAPT: Days 8 to 21 Similar to the induction phase, you may receive RYDAPT following your consolidation chemotherapy treatment. RYDAPT: Days 8 to 21 If you have tested positive for FLT3 and your health care provider has prescribed RYDAPT (midostaurin) capsules, you will receive it following the first 7 days of induction chemotherapy treatment. Please follow your health care provider s instructions for treatment with RYDAPT * The number of induction cycles is based on the clinical trial. Your health care provider may choose to adjust this number depending on your individual treatment plan. In a clinical study, RYDAPT was given on Day 8 of treatment following chemotherapy. IMPORTANT SAFETY INFORMATION (CONTINUED) Taking Other Medicines With RYDAPT Before taking RYDAPT, you should tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. RYDAPT may affect how these medicines work, or these other medicines may affect how RYDAPT works You should also tell your doctor if you are already taking RYDAPT and are prescribed a new medicine that you have not taken previously during RYDAPT treatment A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 7

8 RYDAPT HAS BEEN PROVEN TO HELP PATIENTS WITH NEWLY DIAGNOSED FLT3-POSITIVE AML LIVE LONGER In a clinical trial of adult patients with newly diagnosed FLT3-positive AML: 717 patients were studied to determine whether adding RYDAPT (midostaurin) capsules to chemotherapy would help them live longer 360 patients received RYDAPT plus chemotherapy 357 patients received placebo plus chemotherapy IMPORTANT SAFETY INFORMATION (CONTINUED) Allergic Reactions Do not take RYDAPT if you are allergic to midostaurin or any of the ingredients in RYDAPT If you develop signs of an allergic reaction, seek medical help immediately Signs of an allergic reaction include trouble breathing, flushing, chest pain, throat tightness, and swelling of the face, lips, tongue, or throat 8

9 Patients who received treatment with RYDAPT plus chemotherapy lived longer than patients receiving chemotherapy alone 23% Reduction in the risk of death compared with standard-of-care chemotherapy alone A specific median overall survival (the time point at which half the patients were still alive) could not be reliably estimated. More patients taking RYDAPT (midostaurin) capsules plus chemotherapy (59%) received stem cell transplant (SCT) than patients taking chemotherapy alone (55%). At the time of SCT, study medication was stopped in these patients. In addition, patients who received RYDAPT with chemotherapy in the trial went longer without certain complications (failure to achieve complete remission within 60 days of starting treatment, progression of leukemia, or death) than patients who received chemotherapy alone. Median 8.2 months vs median 3 months IMPORTANT SAFETY INFORMATION (CONTINUED) Pregnancy, Breastfeeding, Fertility RYDAPT should not be used during pregnancy since it may harm an unborn baby If you become pregnant, think you may be, or are planning to be, tell your doctor right away If you are able to become pregnant, your doctor should perform a pregnancy test within 7 days before you start RYDAPT. Effective birth control should be used during treatment and for at least 4 months after the last RYDAPT dose Men taking RYDAPT who have female partners who are able to become pregnant should use effective birth control during treatment with RYDAPT and for at least 4 months after the last dose Pregnancy registry: There is a pregnancy registry that monitors the health of females and their babies exposed to RYDAPT during pregnancy. Females who have taken RYDAPT during pregnancy, or who have been exposed to RYDAPT during pregnancy through a male partner taking RYDAPT, should contact Novartis Pharmaceuticals Corporation at or at Do not breastfeed during treatment with RYDAPT and for at least 4 months after the final dose RYDAPT may impair fertility in males and females. You should discuss this with your doctor before starting treatment A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 9

10 QUESTIONS TO ASK YOUR DOCTOR Communicate regularly with your health care provider so informed decisions can be made regarding your treatment. Following are a few questions that you may want to ask your doctor about treatment. Be sure to bring up any additional questions that may help you better understand how to get the most out of your treatment, as all of your questions are important. What symptoms or side effects should I tell my doctor about right away? How will I know if my treatment with RYDAPT (midostaurin) capsules is working? What if I forget to take RYDAPT? Do I need to change my diet during treatment with RYDAPT? How long will I need to stay on RYDAPT? What should I know about taking RYDAPT while sexually active? What if I m pregnant or lactating, or plan on becoming pregnant? How will taking RYDAPT at home be different than taking it in the hospital? Is monitoring required while I m taking RYDAPT? What if I stop taking RYDAPT? What if I take too much RYDAPT? 10 Please see Important Safety Information throughout this brochure and the Summary of Important Information on pages 19 to 21.

11 TAKING RYDAPT TO TREAT NEWLY DIAGNOSED FLT3-POSITIVE AML A twice-daily oral medicine During your stay in the hospital, clinic staff will give you RYDAPT (midostaurin) capsules at the recommended dose of 50 mg (two 25-mg capsules) twice a day taken 12 hours apart in addition to your regular chemotherapy. RYDAPT comes in a soft, orange capsule and is taken by mouth with food. RYDAPT is only available in 25-mg capsules. Your doctor will likely begin to give you RYDAPT the day after your first 7 days of induction chemotherapy. INDUCTION (1 to 2 cycles) CONSOLIDATION (up to 4 cycles) Chemotherapy Days 1 to 7 Chemotherapy Days 1, 3, and 5 After testing positive for FLT3 RYDAPT 2x/day Days 8 to 21* RYDAPT 2x/day Days 8 to 21* Your doctor may tell you to decrease your dose, temporarily stop, or completely stop taking RYDAPT if you develop certain side effects during treatment with RYDAPT. * In a clinical study, RYDAPT was given on Day 8 of treatment following chemotherapy. IMPORTANT SAFETY INFORMATION (CONTINUED) Lung Problems RYDAPT may cause serious lung problems that may lead to death when used alone or in combination with other chemotherapy medicines If you develop a new or worsening cough, shortness of breath, or chest discomfort, contact your doctor immediately. These may be signs of serious lung problems A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 11

12 TIPS FOR TAKING RYDAPT How to take RYDAPT Always take RYDAPT (midostaurin) capsules exactly as your doctor or pharmacist has told you to. Your doctor or pharmacist will tell you exactly how many capsules of RYDAPT you need to take. Do not change the dose without talking to your doctor. The usual dosage is 50 mg (2 capsules), twice daily (4 capsules total per day) Take RYDAPT every 12 hours and at approximately the same time each day (for example, once in the morning and once in the evening) Swallow RYDAPT capsules whole with a glass of water. Do not open, crush, or chew the capsules. Take RYDAPT with food RYDAPT is administered according to a dosage regimen together with chemotherapy treatments. It is very important to follow your doctor s recommendation If you miss a dose of RYDAPT, take your next dose at your scheduled time. Do not take an extra dose to make up for a missed dose If you vomit after taking a dose of RYDAPT, do not take an extra dose. Take your next dose at your scheduled time IMPORTANT SAFETY INFORMATION (CONTINUED) Side Effects Most common side effects reported during RYDAPT treatment for acute myeloid leukemia (AML) included: low level of white blood cells vomiting nose bleeds with fever (febrile neutropenia) headache device-related infection nausea bruising high blood sugar levels redness, pain, or ulcers inside muscle or bone pain (hyperglycemia) the mouth (mucositis) upper respiratory infection If side effects including nausea, vomiting, and diarrhea occur, get worse, or do not go away during treatment with RYDAPT, you should contact your doctor immediately. Your doctor may need to decrease your dose, temporarily stop, or completely stop your treatment with RYDAPT These are not all of the possible side effects of RYDAPT. For more information, ask your doctor or pharmacist 12

13 TWICE A DAY Two 25-mg capsules 50 mg, 2x/day Taking RYDAPT (midostaurin) capsules at the same time each day will help you remember when to take your medicine. Your doctor may prescribe additional medicines to help with nausea, if necessary. Capsules not actual size It is unknown if RYDAPT is safe and effective in children. A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 13

14 CAREGIVERS PLAY AN ESSENTIAL ROLE IN THE LIVES OF PATIENTS WITH AML When someone learns that they have FLT3-positive AML, there is suddenly a lot of information that needs to be processed, as well as practical considerations about going to the hospital and taking care of things at home. It may be difficult for a patient to absorb all of these details, especially at a time when they re not feeling well and are experiencing a wide range of emotions. Your role as a caregiver is an important one you are not only a source of support and comfort, but also an advocate for your loved one with newly diagnosed FLT3-positive AML. You become their eyes and ears, and are there at doctor s visits to listen and record information about what can be expected during the treatment journey. You are also the person who helps them stay on track with their treatments and medicines and helps them to meet their basic needs. As a caregiver, we understand that you re also dealing with your own emotions, as well as the day-to-day considerations that accompany caring for someone with newly diagnosed FLT3-positive AML. Following are some tips that may be useful while you re helping your loved one through this time. IMPORTANT SAFETY INFORMATION (CONTINUED) Taking Other Medicines With RYDAPT (midostaurin) capsules Before taking RYDAPT, you should tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. RYDAPT may affect how these medicines work, or these other medicines may affect how RYDAPT works You should also tell your doctor if you are already taking RYDAPT and are prescribed a new medicine that you have not taken previously during RYDAPT treatment 14

15 TIPS FOR CAREGIVERS Keep a notebook with treatment-related information. Learn about insurance and/or financial resources that may be available. Try to stay positive. Make time for yourself. Be sure to ask questions and take notes during medical appointments so that you can review them later. Keep track of the contact information for your loved one s health care team, as well as important information about medicines, dosages, symptoms, and side effects of treatments. Navigating insurance issues can be challenging, but it s possible to get help. A doctor and his or her office staff can help you sort out some of the details, and if your loved one is prescribed RYDAPT (midostaurin) capsules, Novartis has a patient support program for eligible patients. Learn more about RYDAPT NOW, brought to you by Novartis Patient Assistance Now Oncology, on the following pages and visit to learn how to enroll. Your outlook can make a big difference in the life of your loved one with newly diagnosed FLT3- positive AML. Of course, remaining positive isn t always easy, so seek out advice and support from family members, friends, professional counselors, and doctors. Patient advocacy groups can put you in touch with others who are going through a similar situation. It can be easy to neglect yourself while you re caring for someone with newly diagnosed FLT3- positive AML. Be sure to continue to eat right and exercise, and take some time to relax. A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 15

16 RYDAPT NOW A PARTNER DURING YOUR JOURNEY Care Coordinators at RYDAPT NOW can help you gain access to your medicine and provide the following support: Assisting you with access to RYDAPT (midostaurin) capsules as you transition between inpatient and outpatient settings Helping you find a pharmacy based on your insurance plan, and get your medicine according to your plan s guidelines Offering support with insurance verification to help you understand how to get your medicine and what your financial responsibilities are Providing information about financial assistance that may be available Providing information about prior authorization requirements Initiating a benefits investigation in the inpatient setting in preparation for transitioning to the outpatient setting Call or visit for information on how to enroll in RYDAPT NOW and to learn more about the ways we can help. Novartis cannot guarantee that patients who enroll in RYDAPT NOW will be successful in obtaining insurance coverage for treatment or will receive financial assistance. 16

17 RYDAPT NOW ACCESS AND FINANCIAL SUPPORT For appropriate patients with AML If your doctor prescribes RYDAPT (midostaurin) capsules for newly diagnosed FLT3+ AML based on the approved label and there is a delay in your insurance coverage as you transition out of the hospital, you may be eligible to receive a free 14-day supply of RYDAPT shipped directly to your home to avoid disruption in your treatment. No purchase of RYDAPT is required. Novartis cannot guarantee that every patient who enrolls in RYDAPT NOW will be successful in obtaining insurance coverage for treatment. Novartis Oncology Universal Co-Pay Program $ 10 CO-PAY You may be eligible for immediate co-pay savings on your next prescription of RYDAPT. Commercially insured patients pay $10 per month Novartis will pay the remaining co-pay, up to $15,000 per calendar year* * Limitations apply. Patient must have commercial insurance. Offer is not valid under Medicare, Medicaid, or any other federal or state program. Novartis reserves the right to rescind, revoke, or amend this program without notice. For full terms and conditions, visit or call IMPORTANT SAFETY INFORMATION (CONTINUED) Allergic Reactions Do not take RYDAPT if you are allergic to midostaurin or any of the ingredients in RYDAPT If you develop signs of an allergic reaction, seek medical help immediately Signs of an allergic reaction include trouble breathing, flushing, chest pain, throat tightness, and swelling of the face, lips, tongue, or throat A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 17

18 HOW TO ENROLL IN RYDAPT NOW OR Access RYDAPT NOW online at and download the Service Request Form Complete the Service Request Form with your health care provider and fax it to Call from 9 to 8 ET, Monday through Friday, to begin the enrollment process RYDAPT NOW Care Coordinators are able to provide information in more than 160 languages. 18

19 SUMMARY OF IMPORTANT INFORMATION What is RYDAPT? RYDAPT (midostaurin) capsules is a prescription medicine used to treat adults: newly diagnosed with a certain type of acute myeloid leukemia (AML), in combination with certain chemotherapy medicines Your health care provider will perform a test to make sure RYDAPT is right for you It is not known if RYDAPT is safe and effective in children. Do not take RYDAPT if you are allergic to midostaurin or any of the ingredients in RYDAPT. See page 21 for a complete list of ingredients in RYDAPT. Signs and symptoms of an allergic reaction to RYDAPT have included trouble breathing, flushing, chest pain, throat tightness, and swelling of your lips, mouth, or throat. Get medical help right away if you have any of these signs or symptoms Before you take RYDAPT, tell your health care provider about all of your medical conditions, including if you: have any lung or breathing problems are pregnant or plan to become pregnant. RYDAPT may cause harm to your unborn baby. Tell your health care provider right away if you become pregnant during treatment with RYDAPT or think you may be pregnant Pregnancy Registry: There is a pregnancy registry that monitors the health of females and their babies exposed to RYDAPT during pregnancy. Females who have taken RYDAPT during pregnancy, or who have been exposed to RYDAPT during pregnancy through a male partner taking RYDAPT, should contact Novartis Pharmaceuticals Corporation at or at If you are able to become pregnant, your health care provider may perform a pregnancy test within 7 days before you start RYDAPT Females who are able to become pregnant should use effective birth control (contraception) during treatment with RYDAPT and for at least 4 months after the last dose of RYDAPT Males who have female partners that are able to become pregnant should use effective birth control during treatment with RYDAPT and for at least 4 months after the last dose of RYDAPT are breastfeeding or plan to breastfeed. It is not known if RYDAPT passes into your breast milk. You should not breastfeed during treatment with RYDAPT and for at least 4 months after the last dose of RYDAPT Tell your health care provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. A GUIDE TO COMBINATION TREATMENT WITH RYDAPT Please see Important Safety Information throughout this brochure. 19

20 SUMMARY OF IMPORTANT INFORMATION (CONTINUED) How should I take RYDAPT? Take RYDAPT (midostaurin) capsules exactly as your health care provider tells you Your health care provider will tell you how many capsules of RYDAPT you need to take. Do not change your dose unless your health care provider tells you to Your health care provider will prescribe medicines to help prevent the nausea and vomiting during treatment with RYDAPT Take RYDAPT 2 times a day (about every 12 hours apart) Take RYDAPT with food Do not open or crush RYDAPT capsules If you miss a dose of RYDAPT, take your next dose at your scheduled time. Do not take an extra dose to make up for a missed dose If you vomit after taking a dose of RYDAPT, do not take an extra dose. Take your next dose at your scheduled time What are the possible side effects of RYDAPT? RYDAPT may cause serious side effects, including: Lung problems. RYDAPT may cause lung problems that may lead to death when used alone or in combination with other chemotherapy medicines. Get medical help right away if you have any new or worsening lung symptoms, including cough, chest discomfort, or shortness of breath The most common side effects of RYDAPT in people with AML include: low white blood cell counts vomiting with fever (febrile neutropenia) headache nausea bruising redness, pain, or ulcers on the muscle or bone pain inside of your mouth (mucositis) nose bleeds device-related infection high blood sugar levels (hyperglycemia) upper respiratory tract infection 20 Please see Important Safety Information throughout this brochure.

21 SUMMARY OF IMPORTANT INFORMATION (CONTINUED) Call or inform your health care provider if nausea, vomiting, or diarrhea occurs, gets worse, or does not go away. RYDAPT (midostaurin) capsules may cause fertility problems in females and males, which may affect your ability to have children. Talk to your health care provider if you have concerns about fertility. Your health care provider may tell you to decrease your dose, temporarily stop, or completely stop taking RYDAPT if you develop certain side effects during treatment with RYDAPT. Your health care provider will do blood tests to check you for side effects during treatment with RYDAPT. These are not all of the possible side effects of RYDAPT. Call your doctor for medical advice about side effects. You may report side effects to FDA at FDA How should I store RYDAPT? Store RYDAPT at room temperature between 68 F to 77 F (20 C to 25 C) Keep RYDAPT in the original package to protect from moisture Keep RYDAPT and all medicines out of the reach of children. General information about the safe and effective use of RYDAPT Medicines are sometimes prescribed for conditions not listed in the Patient Information. Do not take RYDAPT for a condition for which it was not prescribed. Do not give RYDAPT to other people, even if they have the same condition or symptoms you have. It may harm them. You can ask your pharmacist or health care provider for information about RYDAPT that is written for health care professionals. What are the ingredients in RYDAPT? Active ingredient: midostaurin Inactive ingredients: polyoxyl 40 hydrogenated castor oil, gelatin, polyethylene glycol 400, glycerin 85%, dehydrated alcohol, corn oil mono-di-triglycerides, titanium dioxide, vitamin E, ferric oxide yellow, ferric oxide red, carmine, hypromellose 2910, propylene glycol, and purified water A GUIDE TO COMBINATION TREATMENT WITH RYDAPT Please see Important Safety Information throughout this brochure. 21

22 SUMMARY Understanding AML AML is the most common type of acute leukemia affecting adults. In 2017, an estimated 21,380 people in the United States will be diagnosed with AML It s important that you know your FLT3 status, as being FLT3 positive affects what treatment options may be available for you INDICATION for RYDAPT (midostaurin) capsules RYDAPT is an oral prescription medicine used in combination with certain chemotherapy medicines to treat adults with newly diagnosed acute myeloid leukemia (AML) who have a defect in a gene called FLT3. Your doctor will perform a test to make sure RYDAPT is right for you. RYDAPT should not be used alone to induce remission in people with AML. It is unknown if RYDAPT is safe and effective in children. 22 Please see Important Safety Information throughout this brochure and the Summary of Important Information on pages 19 to 21.

23 SUMMARY (CONTINUED) RYDAPT has been proven to help people with newly diagnosed FLT3-positive AML live longer 23% reduction in the risk of death compared with standard-of-care chemotherapy alone A specific median overall survival (the time point at which half the patients were still alive) could not be reliably estimated RYDAPT is a capsule used to treat newly diagnosed FLT3-positive AML The usual dosage is 50 mg (2 capsules), twice daily (4 capsules total per day) Take RYDAPT (midostaurin) capsules every 12 hours and at approximately the same time each day (for example, once in the morning and once in the evening) IMPORTANT SAFETY INFORMATION (CONTINUED) Side Effects Most common side effects reported during RYDAPT treatment for acute myeloid leukemia (AML) included: low level of white blood cells with fever (febrile neutropenia) nausea redness, pain, or ulcers inside the mouth (mucositis) vomiting headache bruising muscle or bone pain nose bleeds device-related infection high blood sugar levels (hyperglycemia) upper respiratory infection If side effects including nausea, vomiting, and diarrhea occur, get worse, or do not go away during treatment with RYDAPT, you should contact your doctor immediately. Your doctor may need to decrease your dose, temporarily stop, or completely stop your treatment with RYDAPT These are not all of the possible side effects of RYDAPT. For more information, ask your doctor or pharmacist RYDAPT may cause harm to your unborn baby. Tell your health care provider right away if you become pregnant during treatment with RYDAPT or think you may be pregnant. See page 19 for additional information RYDAPT may cause lung problems that may lead to death when used alone or in combination with other chemotherapy medicines. Get medical help right away if you have any new or worsening lung symptoms, including cough, chest discomfort, or shortness of breath. See page 20 for additional information A GUIDE TO COMBINATION TREATMENT WITH RYDAPT 23

24 How to enroll in RYDAPT NOW Access RYDAPT NOW online at and download the Service Request Form Complete the Service Request Form with your health care provider and fax it to Call from 9 to 8 ET, Monday through Friday, to begin the enrollment process For information on retail and/or specialty pharmacies that can fill your prescription for RYDAPT, call RYDAPT NOW at OR My LIVING PROOF THAT TREATING NEWLY DIAGNOSED FLT3-POSITIVE AML HAS CHANGED INDICATION for RYDAPT (midostaurin) capsules RYDAPT is an oral prescription medicine used in combination with certain chemotherapy medicines to treat adults with newly diagnosed acute myeloid leukemia (AML) who have a defect in a gene called FLT3. Your doctor will perform a test to make sure RYDAPT is right for you. RYDAPT should not be used alone to induce remission in people with AML. It is unknown if RYDAPT is safe and effective in children. IMPORTANT SAFETY INFORMATION for RYDAPT Allergic Reactions Do not take RYDAPT if you are allergic to midostaurin or any of the ingredients in RYDAPT If you develop signs of an allergic reaction, seek medical help immediately Signs of an allergic reaction include trouble breathing, flushing, chest pain, throat tightness, and swelling of the face, lips, tongue, or throat Please see Important Safety Information throughout this brochure and the Summary of Important Information on pages 19 to % Reduction in the risk of death compared with standardof-care chemotherapy alone A specific median overall survival (the time point at which half the patients were still alive) could not be reliably estimated Novartis Pharmaceuticals Corporation East Hanover, New Jersey Novartis 8/18 MID