For problems of leptin deficiency in people with congenital or acquired generalized lipodystrophy Getting started on MYALEPT (metreleptin) for injection MEET LORELEI, A YOUNG GIRL WITH ACQUIRED GENERALIZED LIPODYSTROPHY. Lorelei has been diagnosed with acquired generalized lipodystrophy and is on MYALEPT treatment. Patients with generalized lipodystrophy experience different problems, and those problems can vary from patient to patient. APPROVED USE MYALEPT is a prescription medicine used with a healthcare provider-recommended diet to treat problems of leptin deficiency (lack of leptin) in people with congenital or acquired generalized lipodystrophy. It is not known if MYALEPT is safe and effective to treat problems caused by partial lipodystrophy or to treat liver disease, including non-alcoholic steatohepatitis (NASH). MYALEPT should not be used to treat people with HIV-related lipodystrophy or people with metabolic disease, including diabetes and high triglycerides, without signs or symptoms of congenital or acquired generalized lipodystrophy. IMPORTANT SAFETY INFORMATION MYALEPT may cause serious side effects, including: risk for loss of natural leptin activity or loss of MYALEPT efficacy due to neutralizing antibodies. Some people who use MYALEPT make antibodies in their blood that may reduce how well the leptin in their body (endogenous) works or how well MYALEPT works. Side effects may include infection, problems with blood sugar (including diabetes), or an increase in the amount of fat in your blood (triglycerides) increased risk of lymphoma (a type of blood cancer) MYALEPT is only available through a restricted program called the MYALEPT Risk Evaluation and Mitigation Strategy (REMS) Program. For more information about the MYALEPT REMS Program go to www.myaleptrems.com or call 1-855-669-2537. Please see Important Safety Information on pages 10 and 11.
For problems of leptin deficiency in people with congenital or acquired generalized lipodystrophy MEET TROY, A TEENAGE BOY WITH ACQUIRED GENERALIZED LIPODYSTROPHY. MYALEPT (metreleptin) for injection and generalized lipodystrophy Your doctor has just prescribed MYALEPT for you. MYALEPT is the first and only FDAapproved treatment for problems of leptin deficiency (lack of leptin) in people with congenital or acquired generalized lipodystrophy. If you have one of these types of generalized lipodystrophy, you understand the difficulties that come with living with such a rare disease. Some of these difficulties may include Dealing with limited information about your disease Managing the financial burden that comes with having a rare disease such as lipodystrophy Having a treatment option may help you manage problems of leptin deficiency associated with generalized lipodystrophy. Before you begin, it s important to Read this entire brochure, including the full Prescribing Information and Medication Guide in the pocket of this brochure Review all materials included in the package containing your MYALEPT (you should have received this package when your doctor prescribed you MYALEPT) If you have questions along the way, talk to your prescribing healthcare provider. Do not use MYALEPT if you have general obesity not caused by a congenital leptin deficiency or are allergic to metreleptin or any of the ingredients in MYALEPT. Information provided in this brochure is not a substitute for talking with your healthcare provider. Your healthcare provider is the best source for information about your disease. Please see Important Safety Information on pages 10 and 11. 2
What s inside What is MYALEPT? How does MYALEPT work? Who should not take MYALEPT? What should I tell my healthcare provider before using MYALEPT? How MYALEPT has been shown to help in a clinical study What is the most important information I should know about MYALEPT? How should I take MYALEPT? When should I take MYALEPT? Important Safety Information Storing and preparing MYALEPT What are the possible side effects of MYALEPT? Tips for taking MYALEPT Notes Assistance with MYALEPT through the BYMYSIDE Patient Support Program 11.3 mg per vial 3
What is MYALEPT (metreleptin) for injection? MYALEPT is a prescription medicine used with a diet recommended by your healthcare provider to treat problems caused by not having enough leptin in your body (leptin deficiency) in people with congenital or acquired generalized lipodystrophy. MYALEPT can help treat some of these problems, such as high levels of sugar and triglyceride fats in the blood. It is not known if MYALEPT is safe and effective when used to treat problems (complications) caused by partial lipodystrophy or to treat liver disease, including NASH. MYALEPT should not be used to treat people with HIV-related lipodystrophy or people with metabolic disease, including diabetes and high triglycerides, without signs or symptoms of congenital or acquired generalized lipodystrophy. You have been prescribed MYALEPT because you have generalized lipodystrophy and it is causing problems of leptin deficiency. People with generalized lipodystrophy often experience problems with leptin deficiency. When used with a diet recommended by your healthcare provider, MYALEPT can treat some of these problems, such as high levels of sugar and triglyceride fats in the blood. How does MYALEPT work? MYALEPT attaches to the leptin receptor in your body and may help improve some of the problems caused by not having enough leptin in the body. MYALEPT is thought to work by helping the body respond better to insulin and by reducing food intake. WHY DOES LEPTIN DEFICIENCY HAPPEN? The loss of body fat that happens in people with generalized lipodystrophy causes a decrease of a hormone in the body called leptin. Please see Important Safety Information on pages 10 and 11. 4
For problems of leptin deficiency in people with congenital or acquired generalized lipodystrophy BETWEEN AGES 4 AND 6, TROY STARTED LOSING THE FAT IN HIS CHEEKS. A FEW YEARS LATER, HE STARTED COMPLETELY LOSING FAT ALL OVER HIS BODY. JASON, TROY S FATHER Who should not take MYALEPT? Do not use MYALEPT if you have general obesity that is not caused by a congenital leptin deficiency or are allergic to metreleptin or any of the ingredients in MYALEPT. What should I tell my healthcare provider before using MYALEPT? Before using MYALEPT, tell your healthcare provider if you have any medical conditions including if you Have or have had problems with your blood cells, including low blood cell counts (especially your white blood cells), bone marrow, immune system, pancreas, swollen lymph nodes, lymphoma, high blood triglyceride levels, or use insulin or a sulfonylurea Are pregnant or plan to become pregnant as it is unknown if MYALEPT will harm your unborn baby. If you become pregnant while using MYALEPT, talk to your healthcare provider about registering with a program to collect information about the outcomes of moms and babies exposed to MYALEPT during pregnancy. You can enroll in the MYALEPT program by calling 1-855-669-2537 Are nursing or plan to nurse. You should not nurse while you take MYALEPT Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. 11.3 mg per vial 5
How MYALEPT (metreleptin) for injection has been shown to help in a clinical study MYALEPT may not work for every person. Individual results may vary. It is important to take MYALEPT as prescribed by your healthcare provider. In the clinical study of patients taking MYALEPT, blood tests were used to track certain problems in 48 patients with generalized lipodystrophy. Some of the patients were tested to measure the amounts of fat and sugar in their blood using A1C, fasting blood sugar, and fasting blood triglyceride lab tests Some patients were also on medications for diabetes and high triglycerides After taking MYALEPT for 1 year, patients in the clinical study experienced improvements in 3 lab tests: A1C 2 % AVERAGE REDUCTION An A1C test provides a 3-month average of your blood sugar levels. (35 patients; combined average A1C starting at 8.5%) Fasting blood sugar 49 mg/dl AVERAGE REDUCTION A fasting blood sugar test measures the amount of sugar, called glucose, from a sample of blood and is typically done when you have not eaten for at least 8 hours (fasting). (37 patients; average fasting blood sugar starting at 174 mg/dl) Fasting blood triglycerides 184 mg/dl MEDIAN REDUCTION a A fasting blood triglycerides test measures the amount of fat in your blood and is typically done when you have not eaten for at least 8 to 12 hours (fasting). (36 patients; median fasting blood triglycerides starting at 348 mg/dl) a Median is a mathematical term that describes the middle number in a list of numbers ordered from lowest to highest. Please see Important Safety Information on pages 10 and 11. 6
For problems of leptin deficiency in people with congenital or acquired generalized lipodystrophy SINCE LORELEI HAS STARTED TAKING MYALEPT, WE VE SEEN IMPROVEMENTS IN HER A1C, FASTING TRIGLYCERIDES, AND GLUCOSE LEVELS. CAMERIN, LORELEI S MOTHER What is the most important information I should know about MYALEPT? MYALEPT may cause serious side effects, including Risk for loss of endogenous leptin activity or loss of MYALEPT efficacy due to neutralizing antibodies. Some people who use MYALEPT make antibodies in their blood that may reduce how well the leptin in your body (endogenous) works or how well MYALEPT works. Side effects may include infection, problems with blood sugar, including diabetes, and/or an increase in the amount of fat in your blood (triglycerides) Lymphoma (a type of blood cancer). There may be an increased risk of getting lymphoma when you use MYALEPT MYALEPT is only available through a restricted program called the MYALEPT Risk Evaluation and Mitigation Strategy (REMS) Program. For more information about the MYALEPT REMS Program go to www.myaleptrems.com or call 1-855-669-2537 If your healthcare provider has prescribed MYALEPT for you or a loved one, he or she has already received the special REMS training and certification. 11.3 mg per vial 7
How should I take MYALEPT (metreleptin) for injection? It is important that you learn how to inject MYALEPT the right way. A healthcare provider should show you how to inject MYALEPT before you use it for the first time. A healthcare provider should also watch you inject your MYALEPT dose the first time you inject it Do not inject MYALEPT until your healthcare provider has shown you the right way to inject it. If you have questions or do not understand the instructions, talk to your healthcare provider or pharmacist Areas on body for injection MYALEPT is only given as a subcutaneous injection, which means it is injected under the skin. Only inject MYALEPT into your stomach, thigh, or upper arm Do not inject MYALEPT into a vein or muscle Please see Important Safety Information on pages 10 and 11. 8
For problems of leptin deficiency in people with congenital or acquired generalized lipodystrophy When should I take MYALEPT? After your healthcare provider has shown you the proper way to prepare and administer your medication, MYALEPT should be self-injected or injected by a caregiver once per day, at the same time of day. Remember to take MYALEPT exactly as your healthcare provider instructs you. If you miss a dose, take it as soon as you remember. Take your regular dose the next day at your normal time. Do not take more than your regular daily dose in a single day. Do not take an extra dose or increase the amount of your dose to make up for a missed dose Do not mix MYALEPT and insulin in the same syringe or vial. Although MYALEPT and insulin doses may be given at the same time, do not inject MYALEPT and insulin in the same injection site Use MYALEPT the right way. For complete instructions, see the Instructions for Use in the pocket of this brochure. DID YOU KNOW? The BYMYSIDE Program offers an optional In-Home Nursing Injection Support Program at no cost to you. Through this program, a registered home health nurse can come to your home to help supervise the preparation and administration of your first dose of MYALEPT. 11.3 mg per vial 9
IMPORTANT SAFETY INFORMATION FOR MYALEPT (METRELEPTIN) FOR INJECTION MYALEPT may cause serious side effects, including: risk for loss of natural leptin activity or loss of MYALEPT efficacy due to neutralizing antibodies. Some people who use MYALEPT make antibodies in their blood that may reduce how well the leptin in their body (endogenous) works or how well MYALEPT works. Side effects may include infection, problems with blood sugar (including diabetes), or an increase in the amount of fat in your blood (triglycerides) increased risk of lymphoma (a type of blood cancer) MYALEPT is only available through a restricted program called the MYALEPT Risk Evaluation and Mitigation Strategy (REMS) Program. For more information about the MYALEPT REMS Program go to www.myaleptrems.com or call 1-855-669-2537. Do not use MYALEPT if you have general obesity not caused by a congenital leptin deficiency or are allergic to metreleptin or any of the ingredients in MYALEPT. Before using MYALEPT, tell your healthcare provider if you have any medical conditions including if you: have or have had problems with your blood cells, including low blood cell counts (especially your white blood cells), bone marrow, immune system, pancreas, swollen lymph nodes, lymphoma, high blood triglyceride levels, or use insulin or a sulfonylurea are pregnant or plan to become pregnant as it is unknown if MYALEPT will harm your unborn baby. If you become pregnant while using MYALEPT, talk to your healthcare provider about registering with a program to collect information about the outcomes of moms and babies exposed to MYALEPT during pregnancy. You can enroll in the MYALEPT program by calling 1-855-669-2537 are nursing or plan to nurse. You should not nurse while you take MYALEPT Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Take MYALEPT exactly as your healthcare provider instructs you. For newborns and infants, mix MYALEPT with sterile water for injection (preservative-free) (WFI). MYALEPT contains benzyl alcohol when mixed with a liquid called bacteriostatic water for injection (BWFI). Serious side effects including death have happened in newborns or infants who have received the preservative benzyl alcohol. 10
Low blood sugar (hypoglycemia) can occur when MYALEPT is used with medicines that can cause hypoglycemia, such as insulin or sulfonylurea. Your insulin or sulfonylurea dose may need to be lowered while you use MYALEPT. Signs and symptoms of low blood sugar may include shakiness, sweating, headache, drowsiness, weakness, dizziness, confusion, irritability, hunger, fast heart beat, and/or feeling jittery. Talk to your doctor about the symptoms and treatment for hypoglycemia. Worsening symptoms of autoimmune disease may occur in people who have or have had certain problems with their immune system. Talk to your doctor about what symptoms you should watch for that may require further testing. Allergic reactions (hypersensitivity), including serious allergic reactions, can happen in people who use MYALEPT. Call your healthcare provider right away if you have any signs or symptoms of an allergic reaction including swelling of the face, lips, tongue or throat; problems breathing or swallowing; rash; itching (hives); fainting or feeling dizzy; or very rapid heartbeat. The most common side effects of MYALEPT include headache, low blood sugar (hypoglycemia), decreased weight, and/or abdominal pain. Talk to your healthcare provider about any side effect that bothers you or that does not go away. APPROVED USE MYALEPT is a prescription medicine used with a healthcare provider-recommended diet to treat problems of leptin deficiency (lack of leptin) in people with congenital or acquired generalized lipodystrophy. It is not known if MYALEPT is safe and effective to treat problems caused by partial lipodystrophy or to treat liver disease, including non-alcoholic steatohepatitis (NASH). MYALEPT should not be used to treat people with HIV-related lipodystrophy or people with metabolic disease, including diabetes and high triglycerides, without signs or symptoms of congenital or acquired generalized lipodystrophy. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. 11.3 mg per vial 11
For problems of leptin deficiency in people with congenital or acquired generalized lipodystrophy MY FAVORITE SUBJECTS IN SCHOOL ARE MATH AND ENGLISH LANGUAGE DEVELOPMENT. LORELEI Storing and preparing MYALEPT (metreleptin) for injection Your healthcare provider will tell you if MYALEPT should be mixed with a liquid called sterile water for injection (WFI) (preservative-free) or with bacteriostatic water for injection (BWFI). In adult and pediatric patients, mix with BWFI MYALEPT mixed with BWFI can be used for more than 1 dose for up to 3 days when stored in the refrigerator between 36 F and 46 F (2 C and 8 C) in their cartons and out of the light. After mixing, the MYALEPT liquid in the vial should be clear and colorless. Throw away any unused MYALEPT after 3 days. In newborns or infants, mix with WFI MYALEPT mixed with WFI should be used right away. After mixing, the MYALEPT liquid in the vial should be clear and colorless. Throw away any unused MYALEPT; it cannot be saved for later use. Important preparation and storage information Do not shake MYALEPT after mixing. Do not freeze MYALEPT before or after mixing. Do not use MYALEPT past the expiration date printed on the vial. Do not use if the white powder in the vial is discolored, cloudy, or has any lumps or particles in it. Throw the vial away and get a new one. Serious side effects including death have happened in newborns or infants who have received the preservative benzyl alcohol. MYALEPT, when mixed with BWFI, contains benzyl alcohol. MYALEPT, when mixed with WFI (preservative-free), contains no benzyl alcohol. When MYALEPT is used in newborns or infants, MYALEPT should be mixed with WFI. Do not suddenly stop using MYALEPT as it may cause a serious problem with your pancreas and very high triglycerides. Please see Important Safety Information on pages 10 and 11. 12
What are the possible side effects of MYALEPT? Look out for these signs and symptoms: See What is the most important information I should know about MYALEPT? on page 7. Low blood sugar (hypoglycemia). You may get low blood sugar if you use MYALEPT with another medicine that can cause low blood sugar, such as insulin or sulfonylurea. The dose of your insulin or sulfonylurea may need to be lowered while you use MYALEPT. Signs and symptoms of low blood sugar may include Shakiness Drowsiness Confusion Fast heartbeat Sweating Weakness Irritability Feeling jittery Headache Dizziness Hunger Talk with your healthcare provider about how to recognize and treat low blood sugar. Make sure that your family and other people around you a lot know how to recognize and treat low blood sugar. Autoimmunity. People who have or have had certain problems with their immune system (autoimmune disease) may have worsening of their symptoms with MYALEPT. Talk to your healthcare provider about what symptoms you should watch for that would warrant further testing. Allergic reactions (hypersensitivity). Allergic reactions can happen in people who use MYALEPT. Talk to your healthcare provider right away if you have any symptoms of an allergic reaction including swelling of your face, lips, tongue, or throat; problems breathing or swallowing; severe rash or itching; fainting or feeling dizzy; or very rapid heartbeat. See Who should not take MYALEPT? on page 5. Benzyl alcohol toxicity. Serious side effects including death have happened in newborns or infants who have received the preservative benzyl alcohol. MYALEPT, when mixed with a liquid called bacteriostatic water for injection (BWFI), contains benzyl alcohol. MYALEPT, when mixed with sterile water for injection (WFI) (preservative-free), contains no benzyl alcohol. When MYALEPT is used in newborns or infants, MYALEPT should be mixed with WFI. The most common side effects of MYALEPT include headache, low blood sugar (hypoglycemia), decreased weight, and abdominal pain. Talk to your healthcare provider about any side effect that bothers you or that does not go away. These are not all the side effects with MYALEPT. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. 11.3 mg per vial 13
Tips for taking MYALEPT (metreleptin) for injection Try these tips to help you prepare for your injection with MYALEPT: Know the steps in the process. To get to know the steps, read this brochure and the Instructions for Use booklet provided to you with your MYALEPT package ahead of time. It may help to read the steps aloud. If you have questions about how to inject your medication properly, make sure to ask your healthcare provider. Find a place that is clean and well lit where you can do the injection. Ask for help. You may feel more comfortable if a care partner does the injection for you. Put ice on the injection site. You may want to use ice at the injection site after your injection to help lessen some of the pain you might feel. Make sure the vial with powder (MYALEPT) is at room temperature before you mix and inject it. Take a vial with powder out of the refrigerator 10 to 15 minutes before you plan to mix it. by side Support Access Resources Visit myaleptbymyside.com or call 1-855-669-2537, from 8 AM to 8 PM (ET), Monday through Friday, to speak with a member of the BYMYSIDE team. Please see Important Safety Information on pages 10 and 11. 14
For problems of leptin deficiency in people with congenital or acquired generalized lipodystrophy SOME OF MY FAVORITE THINGS ARE BASKETBALL, LIONS, DRAGONS, AND DINOSAURS. LORELEI Notes 11.3 mg per vial 15
by side Support Access Resources The BYMYSIDE team is available to answer any questions you may have about getting started with MYALEPT (metreleptin) for injection. A member of the BYMYSIDE team will Work with you to navigate MYALEPT access and affordability options Provide you with support for MYALEPT preparation and administration Coordinate your MYALEPT shipment with the specialty pharmacy Benefits offered through the BYMYSIDE Program Step-by-step training on MYALEPT reconstitution and self-injection Registered home health nurse MYALEPT administration assistance and supervision Helpful tips for staying on treatment and maintaining a regular dosing schedule MYALEPT resources including the Prescribing Information, Medication Guide, and Instructions for Use To learn more, visit myaleptbymyside.com or call 1-855-669-2537, from 8 AM to 8 PM (ET), Monday through Friday, to speak with a member of the BYMYSIDE team. Please see Important Safety Information on pages 10 and 11. 2015 Aegerion Pharmaceuticals, Inc. MYALEPT is a registered trademark and BYMYSIDE is a trademark of Aegerion Pharmaceuticals, Inc. All rights reserved. MYA/US/055 9-15. 11.3 mg per vial