Your guide. to iron chelation therapy with FERRIPROX (deferiprone) 500 mg tablets for oral use. What is FERRIPROX (deferiprone)?

Transcription

1 What is FERRIPROX (deferiprone)? Your guide to iron chelation therapy with FERRIPROX (deferiprone) 500 mg tablets for oral use FERRIPROX is a prescription medicine used to treat people with thalassemia (thal-uh-see-mee-uh) syndromes who have iron overload from blood transfusions, when current iron removal (chelation) therapy does not work well enough. FERRIPROX was approved based on its ability to lower iron levels in the blood. There are no studies to show that FERRIPROX improves the symptoms of the disease, or your ability to function, or that it increases the length of time you may live. It is not known if FERRIPROX tablets are safe and effective to treat iron overload due to blood transfusions in people with any other type of anemia that is long-lasting, or in children. What is the most important information I should know about FERRIPROX? FERRIPROX can cause serious side effects, including a very low white blood cell count in your blood. One type of white blood cell that is important for fighting infections is called a neutrophil (NEWT-ro-fill). A low neutrophil count, otherwise known as neutropenia (NEWTro-pee-nee-uh), can put you at risk of infection. Neutropenia is common with FERRIPROX and can become severe in some patients. Severe neutropenia is known as agranulocytosis (ay-gran-you-lowsigh-tow-sis). If you develop agranulocytosis you will be at risk of developing serious infections that can lead to death. Your healthcare provider should do a blood test before you start FERRIPROX and weekly during treatment to check your neutrophil count. If you develop neutropenia, your healthcare provider should check your blood counts every day until your white blood cell count improves. Stop taking FERRIPROX and get medical help right away if you develop any of these symptoms of infection: fever sore throat or mouth sores flu-like symptoms chills and severe shaking Please refer to the additional Important Safety Information about FERRIPROX throughout this guide and see Full Prescribing Information, including Important WARNING and Medication Guide available here and on

2 Table of contents Living with thalassemia... Living with thalassemia Thalassemia is an inherited type of blood disease that affects your body s ability to transport oxygen. The trouble with blood transfusions... What is iron chelation therapy?... What is FERRIPROX (deferiprone)?... Who should not take FERRIPROX?... How should I take FERRIPROX?... How do we know when FERRIPROX is working?... What is the most important information I should know about FERRIPROX?... What are other possible side effects of FERRIPROX?... What should I tell my healthcare provider before taking FERRIPROX?... Like anyone else, there is a great deal that you can do to help improve your well-being: you can stay physically active, eat a healthy diet and avoid smoking. You should also take your medications as prescribed by your healthcare provider and remember not to skip doses. You ll also need to keep up with your medical tests and see your doctor regularly. Speak to your healthcare team about the many things you can do to help stay healthy. -month treatment tracker... 5 Patient resources... 8 With the new day comes new strength and new thoughts. Eleanor Roosevelt Kind permission for the following quotes is acknowledged: Page : Quote from Eleanor Roosevelt; Page : Quote from Helen Keller, Copyright American Foundation for the Blind, Helen Keller Archives.

3 The trouble with blood transfusions The oxygen that we breathe is carried around our body by a molecule called hemoglobin (HEE-mow-glow-bin). We all produce hemoglobin, which is found in red blood cells traveling through our bloodstream. However, because of small changes in the genes of people with thalassemia, their hemoglobin can t carry enough oxygen to meet their bodies needs. (For more information on thalassemia, please consult your healthcare provider.) People with thalassemia may need transfusions of healthy red blood cells every two to five weeks. The goal of these transfusions is to maintain a close-to-normal amount of hemoglobin in your blood so that your body gets the oxygen it needs. Unfortunately, your body has no way to get rid of the iron that comes with all the transfused red blood cells. This means that iron builds up in your body over time, causing iron overload. While healthy people store around grams of iron in their body, thalassemia patients without proper treatment can accumulate up to 70 grams of iron by their thirtieth birthday. How many blood transfusions may put me at risk of iron overload? Iron begins to accumulate in the body when a person with thalassemia starts getting regular transfusions, but it may take years before there are physical signs of iron overload. What is iron chelation therapy? Your body has a very limited ability to remove excess iron without help. An iron chelator (KEY-lay-ter) is a medicine that binds to iron and allows the excess to be excreted. To find out more about iron chelation contact Cooley s Anemia Foundation at What is FERRIPROX (deferiprone)? FERRIPROX, available as 500 mg tablets for oral use, is a prescription medicine used to treat people with thalassemia syndromes who have iron overload from blood transfusions, when current iron removal (chelation) therapy does not work well enough. FERRIPROX was approved based on its ability to lower iron levels in the blood. There are no studies to show that FERRIPROX improves the symptoms of the disease, or your ability to function, or that it increases the length of time you may live. It is not known if FERRIPROX tablets are safe and effective to treat iron overload due to blood transfusions in people with any other type of anemia that is long-lasting, or in children. Who should not take FERRIPROX? Do not take FERRIPROX if you are allergic to deferiprone or any of the ingredients in FERRIPROX. You should only take FERRIPROX exactly as prescribed by your healthcare provider, and never change your dose unless otherwise instructed.

4 How should I take FERRIPROX? The recommended initial dose of FERRIPROX is 5 mg/kg, orally, three times per day for a total of 75 mg/kg/day. The maximum recommended dose is mg/kg, three times per day for a total of 99 mg/kg/day. Of course, your healthcare provider will tell you how many FERRIPROX tablets to take. Here are a few helpful guidelines: Since FERRIPROX is taken three times each day, aim to take your first dose in the morning, the second dose at midday, and the third dose in the evening Although you can take FERRIPROX with or without food, taking FERRIPROX with meals may help reduce nausea Allow at least a -hour interval between FERRIPROX and other medications or supplements containing iron, aluminum, or zinc If you take too much FERRIPROX, call your healthcare provider What if I miss a dose? If you miss a dose, take it as soon as you remember. If it is almost time for your next dose, skip the missed dose and then continue with your regular schedule. Do not try to catch up or take two doses at the same time to make up for a missed dose. Remember: It s important to take your iron chelation therapy as prescribed by your doctor. Take an active role in your health You can keep track of the results of your treatment and inform your healthcare provider about any changes you may have noticed. You may wish to use the Treatment Tracker on pages 5 through 7 of this booklet. -month treatment tracker part Baseline Month 9 Month 0 week week week week week week week week FERRIPROX (deferiprone) dose (mg/kg/day) Hemoglobin White blood cell count Neutrophil count Platelet count Medications Serum Ferritin Other iron measures* Transfusions (Circle the number of bags of red blood cells you receive) *How your iron stores will be measured will depend on your healthcare provider. FERRIPROX Patient Information Booklet How do we know when FERRIPROX is working? Your doctor will do a blood test every two to three months to measure the amount of iron in your blood (called serum ferritin (FAIR-it-in) to learn if and how well FERRIPROX is working. In addition, some doctors may measure T* (TEE-too-star), which is an MRI (magnetic resonance imaging) method used to look at iron in your heart, however, the clinical significance of changes in T* values during FERRIPROX treatment is unknown. What is the most important information I should know about FERRIPROX? FERRIPROX can cause serious side effects, including a very low white blood cell count. One type of white blood cell, called a neutrophil, is important for fighting infections. If you have a low neutrophil count (called neutropenia) you may be at risk of developing a serious infection that can lead to death. Neutropenia is common with FERRIPROX and can become severe in some patients. Severe neutropenia is known as agranulocytosis and if you develop agranulocytosis, you will be at risk of developing serious infections that can lead to death. Your healthcare provider should do a blood test before you start FERRIPROX and weekly during treatment to check your neutrophil count. If you develop neutropenia, your healthcare provider should check your blood counts every day until your white blood cell count improves. Stop taking FERRIPROX and get medical help right away if you develop symptoms of an infection such as, fever, sore throat or mouth sores, flu-like symptoms, or chills and severe shaking.

5 What are other possible side effects of FERRIPROX? FERRIPROX can also cause increased liver enzymes in your blood. Your healthcare provider should do a monthly blood test to check your liver function during treatment. Your doctor should tell you about symptoms and signs of an irregular heart rhythm (arrhythmia). It is not clear whether FERRIPROX causes arrhythmia, but it is important that you get medical help right away if you have any of the following symptoms: irregular or fast heart beat lightheadedness or fainting dizziness seizure The most common side effects of FERRIPROX include: reddish-brown-colored urine; this is not harmful and is expected when you are taking FERRIPROX nausea vomiting stomach-area (abdominal) pain joint pain low neutrophil count What should I tell my healthcare provider before taking FERRIPROX? Tell your healthcare provider if you have liver, kidney, or heart problems, especially a heart rhythm problem called QT prolongation or long QT syndrome; have been told that you have low levels of potassium or magnesium in your blood; are pregnant or plan to become pregnant; or have any other medical conditions. FERRIPROX can harm your unborn baby so avoid getting pregnant while on therapy. It is not known if FERRIPROX passes into breast milk. You and your healthcare provider should decide if you will take FERRIPROX or breastfeed since you should not do both. Before taking FERRIPROX tell your healthcare provider if you take other medicines that can lower your neutrophil count or antacids or mineral supplements that contain iron, aluminum, or zinc. Allow at least hours between taking FERRIPROX and products that contain iron, aluminum, or zinc. Notes: Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of FERRIPROX. For more information, ask your healthcare provider or pharmacist. You can find the Full Prescribing Information here and on Optimism is the faith that leads to achievement. Helen Keller Please see Full Prescribing Information, including Important WARNING and Medication Guide available here and on

6 FERRIPROX (deferiprone) Treatment Tracker -month treatment tracker (part of ) Baseline Month Month Month Month FERRIPROX (deferiprone) dose (mg/kg/day) Hemoglobin White blood cell count Neutrophil count Platelet count Medications Serum ferritin Other iron measures* Transfusions (Circle the number of bags of red blood cells you receive) *How your iron stores will be measured will depend on your healthcare provider. Please see Indication and Important Safety Information in this booklet. 5

7 FERRIPROX (deferiprone) Treatment Tracker -month treatment tracker (part of ) Baseline Month 5 Month 6 Month 7 Month 8 FERRIPROX (deferiprone) dose (mg/kg/day) Hemoglobin White blood cell count Neutrophil count Platelet count Medications Serum ferritin Other iron measures* Transfusions (Circle the number of bags of red blood cells you receive) *How your iron stores will be measured will depend on your healthcare provider. Please see Indication and Important Safety Information in this booklet. 6

8 FERRIPROX (deferiprone) Treatment Tracker -month treatment tracker (part of ) Baseline Month 9 Month 0 Month Month FERRIPROX (deferiprone) dose (mg/kg/day) Hemoglobin White blood cell count Neutrophil count Platelet count Medications Serum ferritin Other iron measures* Transfusions (Circle the number of bags of red blood cells you receive) *How your iron stores will be measured will depend on your healthcare provider. Please see Indication and Important Safety Information in this booklet. 7

9 Patient resources FERRIPROX Total Care Partners in patient care FERRIPROX Total Care is a comprehensive patient support program that can answer any questions you may have regarding access to FERRIPROX. Simply call one number, , anywhere in the United States to contact your FERRIPROX Total Care team. FERRIPROX Total Care is administered by Dohmen Life Science Services, a fully integrated specialty pharmacy and patient service company. The dedicated healthcare team for FERRIPROX Total Care includes pharmacists, customer service and reimbursement support specialists, and nursing assistance for appeals and case management. The team can guide you through the process of getting started on FERRIPROX. Go to for more information about the program. FERRIPROX Total Care makes a difference. Cooley s Anemia Foundation Cooley s Anemia Foundation is dedicated to serving people with various forms of thalassemia with the goal of advancing treatment, and educating the medical profession, patients, and the public about thalassemia. Important phone numbers FERRIPROX Total Care: ApoPharma adverse events reporting and medical information requests: US Food and Drug Administration (FDA): -800-FDA-088 Cooley s Anemia Foundation: FERRIPROX Consumer Indication and Important Safety Information What is FERRIPROX (deferiprone)? FERRIPROX is a prescription medicine used to treat people with thalassemia syndromes who have iron overload from blood transfusions, when current iron removal (chelation) therapy does not work well enough. FERRIPROX was approved based on its ability to lower iron levels in the blood. There are no studies to show that FERRIPROX improves the symptoms of the disease, or your ability to function, or that it increases the length of time you may live. It is not known if FERRIPROX tablets are safe and effective to treat iron overload due to blood transfusions in people with any other type of anemia that is long-lasting, or in children. Important Safety Information What is the most important information I should know about FERRIPROX? FERRIPROX can cause serious side effects, including a very low white blood cell count. One type of white blood cell, called a neutrophil, is important for fighting infections. If you have a low neutrophil count (called neutropenia) you may be at risk of developing a serious infection that can lead to death. Neutropenia is common with FERRIPROX and can become severe in some patients. Severe neutropenia is known as agranulocytosis and if you develop agranulocytosis, you will be at risk of a serious infection that can lead to death. Your healthcare provider should do a blood test before you start FERRIPROX and weekly during treatment to check your neutrophil count. If you develop neutropenia, your healthcare provider should check your blood counts every day until your white blood cell count improves. Stop taking FERRIPROX and get medical help right away if you develop symptoms of an infection such as fever, sore throat or mouth sores, flu-like symptoms, or chills and severe shaking. 8

10 Who should not take FERRIPROX? Do not take FERRIPROX if you are allergic to deferiprone or any other ingredients in FERRIPROX. What should I tell my healthcare provider before taking FERRIPROX? Tell your healthcare provider if you have liver, kidney, or heart problems, especially a heart rhythm problem called QT prolongation or long QT syndrome; have been told that you have low levels of potassium or magnesium in your blood; are pregnant or plan to become pregnant; or have any other medical conditions. FERRIPROX can harm your unborn baby so avoid getting pregnant while on therapy. It is not known if FERRIPROX passes into breast milk. You and your healthcare provider should decide if you will take FERRIPROX or breastfeed since you should not do both. Before taking FERRIPROX tell your healthcare provider if you take other medicines that can lower your neutrophil count or antacids or mineral supplements that contain iron, aluminum, or zinc. Allow at least hours between taking FERRIPROX and products that contain iron, aluminum, or zinc. Guidance on FERRIPROX (deferiprone) Total Care FERRIPROX Total Care Partners in patient care FERRIPROX Total Care is a comprehensive patient support program that can answer any questions you may have regarding access to FERRIPROX. Simply call one number, , anywhere in the United States to contact your FERRIPROX Total Care team. FERRIPROX Total Care is administered by Dohmen Life Science Services, a fully integrated specialty pharmacy and patient service company. The dedicated healthcare team for FERRIPROX Total Care includes pharmacists, customer service and reimbursement support specialists, and nursing assistance for appeals and case management. The team can guide you through the process of getting started on FERRIPROX. Go to for more information about the program. FERRIPROX Total Care makes a difference. What are other possible side effects of FERRIPROX? FERRIPROX can cause other serious side effects including increased liver enzyme levels in your blood. Your healthcare provider should do a monthly blood test to check your liver function during treatment. Get medical help right away if you experience irregular or fast heart beat, dizziness, lightheadedness or fainting, or seizure, as this may be a sign of an irregular heart rhythm (arrhythmia). The most common side effects of FERRIPROX are: reddish-brown-colored urine, nausea, vomiting, stomach-area (abdominal) pain, joint pain, and a low neutrophil count. Please see Full Prescribing Information, including Important WARNING and Medication Guide available here and on FERRIPROX (deferiprone) is available in 500 mg tablets, for oral use. Please see Indication and Important Safety Information on page. 9 Ferri-06-0