OVERVIEW
Which treatment is right for you?
It is important to consider many factors, including your age, family planning, personal circumstances, and conversations with your doctor, when choosing treatment.
You have options when it comes to therapies that work to treat Gaucher disease type 1 (GD1). Most adults with GD1 are eligible for oral therapy.
ERT=enzyme replacement therapy; SRT=substrate reduction therapy.
Talk with your doctor about which treatment option may be right for you and your lifestyle.
SHAUNA'S STORY
Hear from a real Cerdelga patient, Shauna, as she talks about her own journey
Shauna talks about her own journey—from struggling with getting the correct diagnosis of GD1 to starting ERT treatment—and then switching to Cerdelga.
Patient stories reflect the real-life experiences of persons diagnosed with GD1 who have been prescribed Cerdelga. However, individual experiences may vary. Patient stories are not necessarily representative of what another person using Cerdelga may experience. This patient was compensated for their time creating this video.
Your healthcare professional is the best source of information regarding your health. Please consult your healthcare professional if you have any questions about your health or treatment.
My name is Shauna Mangum. I was diagnosed with Gaucher disease in July of 1995.
Experiences from as far back as I can remember were nosebleeds, that was the major thing that comes to mind, they indicated there was something not right. I would have nosebleeds that would last several hours, they were frequent, sometimes every day. I also had bone pains which was dismissed as growing pains. In 1990 I was hospitalized for a severe bleeding episode. They immediately called a hematologist because the lab had notified my doctor. So I was moved over to another hospital and seen by a hematologist right away who immediately admitted me into the hospital. I had a platelet count of 11,000. He said I had an enlarged spleen, and he was concerned that I could have leukemia. That took 5 years for a diagnosis.
Yeah, I feel like I’ve given a second chance at experiencing what life has to offer. I began enzyme replacement therapy in March of 2000. It was exciting at first, but the time, very time consuming spending 3 to 4 hours getting an IV every 2 weeks for a busy mom with young children. It’s hard to find a babysitter, or you take the kids with you and hope that they don’t run all over the infusion site destroying things.
It’s extremely important to have a good relationship with a physician that is knowledgeable about Gaucher disease. Together we discuss challenges, treatments, complex issues that they may want to address.
Currently I take Cerdelga. Cerdelga is an oral treatment, and I take a capsule in the morning and a capsule at night, every day. I have more time for myself. I don’t have to schedule ERT. I don’t have to know that I’ve got those 3 hours that I have to commit to ERT every 2 weeks. I don’t have to do that anymore. It’s just become part of my routine, I, all I have to do is take a pill and go on my way.
Today I feel really good, I am much more engaged in many more activities in my life than I ever thought I would be. I continue to run. I’ve run 2 full marathons, 2 half marathons. I run because I enjoy running. My kids are busy, and they keep me going. I work full time.
Talk with your doctor to find out if Cerdelga is right for you.
You may also go to www.Cerdelga.com for the full Prescribing Information including the Patient Medication Guide, or call Genzyme Corporation at 1-800-745-4447.
Indication:
CERDELGA (eliglustat) is a prescription medication used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. Your doctor will perform a test to make sure that CERDELGA is right for you.
Limitations of Use:
CYP2D6 ultra-rapid metabolizers may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect.
A specific dose cannot be recommended for CYP2D6 indeterminate metabolizers.
Important Safety Information
Certain patients should not use CERDELGA based on their CYP2D6 metabolizer status due to an increased risk of side effects, including heart problems. Do not use CERDELGA if you are:
An Extensive Metabolizer (EM) taking a medicine that is a strong or moderate CYP2D6 inhibitor along with another medicine that is a strong or moderate CYP3A inhibitor, an EM with moderate or severe liver problems, or an EM with mild liver problems and taking a medicine that is a strong or moderate CYP2D6 inhibitor.
An Intermediate Metabolizer (IM) taking a medicine that is a strong or moderate CYP2D6 inhibitor along with another medicine that is a strong or moderate CYP3A inhibitor, an IM taking a medicine that is a strong CYP3A inhibitor, or an IM with any degree of liver problems.
A Poor Metabolizer (PM) taking a medicine that is a strong CYP3A inhibitor, or a PM with any degree of liver problems.
Your doctor will perform a test to help determine if CERDELGA is right for you.
CERDELGA can affect the way other medicines work and other medicines can affect how CERDELGA works. Using CERDELGA with other medicines or herbal supplements may cause an increased risk of side effects, including changes in electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Especially tell your doctor if you take St. John's Wort, or medicines for fungal infections, tuberculosis, seizures, heart conditions, high blood pressure, or depression or other mental health problems. Your doctor may need to prescribe a different medicine, change your dose of other medicines, or change your dose of CERDELGA. Tell your doctor about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements before you start taking them.
Before taking CERDELGA, tell your doctor about all of your medical conditions, including heart problems (including a condition called long QT syndrome), a history of heart attack, kidney or liver problems. If you are pregnant or plan to become pregnant or breastfeed, talk to your doctor. It is not known if CERDELGA will harm your unborn baby. Talk to your doctor if you are breastfeeding or planning to breastfeed. It is not known if CERDELGA passes into your breast milk. You and your doctor will decide if you should take CERDELGA or breastfeed. You should not do both.
CERDELGA, used with certain other medications, may cause changes in the electrical activity of your heart (ECG changes) and irregular heartbeat (arrhythmias). Tell your doctor if you have new symptoms such as palpitations, fainting, or dizziness.
The most common side effects (≥10%) of CERDELGA include: tiredness, headache, nausea diarrhea, back pain, pain in extremities, and upper abdominal pain. Call your doctor for medical advice about adverse effects.
Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of CERDELGA. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
It is not known if CERDELGA is safe and effective in children.
Please see the full Prescribing Information, including the Patient Medication Guide, for CERDELGA.
Ready to start or switch to Cerdelga?
Want to know more about how Cerdelga works?
This video explains how Cerdelga works in your body to treat GD1.
Indication
CERDELGA is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. Your doctor will perform a test to make sure that CERDELGA is right for you.
Limitations of Use:
- CYP2D6 ultra-rapid metabolizers may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect.
- A specific dose cannot be recommended for CYP2D6 indeterminate metabolizers.
Important Safety Information
Certain patients should not use CERDELGA based on their CYP2D6 metabolizer status due to an increased risk of side effects, including heart problems. Do not use CERDELGA if you are:
- An Extensive Metabolizer (EM) taking a medicine that is a strong or moderate CYP2D6 inhibitor along with another medicine that is a strong or moderate CYP3A inhibitor, an EM with moderate or severe liver problems, or an EM with mild liver problems and taking a medicine that is a strong or moderate CYP2D6 inhibitor.
- An Intermediate Metabolizer (IM) taking a medicine that is a strong or moderate CYP2D6 inhibitor along with another medicine that is a strong or moderate CYP3A inhibitor, an IM taking a medicine that is a strong CYP3A inhibitor, or an IM with any degree of liver problems.
- A Poor Metabolizer (PM) taking a medicine that is a strong CYP3A inhibitor, or a PM with any degree of liver problems.
Your doctor will perform a test to help determine if CERDELGA is right for you.
CERDELGA can affect the way other medicines work and other medicines can affect how CERDELGA works. Using CERDELGA with other medicines or herbal supplements may cause an increased risk of side effects, including changes in electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Especially tell your doctor if you take St. John's Wort, or medicines for fungal infections, tuberculosis, seizures, heart conditions, high blood pressure, or depression or other mental health problems. Your doctor may need to prescribe a different medicine, change your dose of other medicines, or change your dose of CERDELGA. Tell your doctor about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements before you start taking them.
Before taking CERDELGA, tell your doctor about all of your medical conditions, including heart problems (including a condition called long QT syndrome), a history of heart attack, kidney or liver problems. If you are pregnant or plan to become pregnant or breastfeed, talk to your doctor. It is not known if CERDELGA will harm your unborn baby. Talk to your doctor if you are breastfeeding or planning to breastfeed. It is not known if CERDELGA passes into your breast milk. You and your doctor will decide if you should take CERDELGA or breastfeed. You should not do both.
CERDELGA, used with certain other medicines, may cause changes in the electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Tell your doctor if you have new symptoms such as palpitations, fainting, or dizziness.
The most common side effects (≥10%) of CERDELGA include: tiredness, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain. Call your doctor for medical advice about adverse effects.
Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of CERDELGA. Call your doctor for medical advice about side effects.
It is not known if CERDELGA is safe and effective in children.
Please see the full Prescribing Information, including the Patient Medication Guide, for CERDELGA.
Indication
CERDELGA is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test. Your doctor will perform a test to make sure that CERDELGA is right for you.
Limitations of Use:
- CYP2D6 ultra-rapid metabolizers may not achieve adequate concentrations of CERDELGA to achieve a therapeutic effect.
- A specific dose cannot be recommended for CYP2D6 indeterminate metabolizers.
Important Safety Information
Certain patients should not use CERDELGA based on their CYP2D6 metabolizer status due to an increased risk of side effects, including heart problems. Do not use CERDELGA if you are:
- An Extensive Metabolizer (EM) taking a medicine that is a strong or moderate CYP2D6 inhibitor along with another medicine that is a strong or moderate CYP3A inhibitor, an EM with moderate or severe liver problems, or an EM with mild liver problems and taking a medicine that is a strong or moderate CYP2D6 inhibitor.
- An Intermediate Metabolizer (IM) taking a medicine that is a strong or moderate CYP2D6 inhibitor along with another medicine that is a strong or moderate CYP3A inhibitor, an IM taking a medicine that is a strong CYP3A inhibitor, or an IM with any degree of liver problems.
- A Poor Metabolizer (PM) taking a medicine that is a strong CYP3A inhibitor, or a PM with any degree of liver problems.
Your doctor will perform a test to help determine if CERDELGA is right for you.
CERDELGA can affect the way other medicines work and other medicines can affect how CERDELGA works. Using CERDELGA with other medicines or herbal supplements may cause an increased risk of side effects, including changes in electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Especially tell your doctor if you take St. John's Wort, or medicines for fungal infections, tuberculosis, seizures, heart conditions, high blood pressure, or depression or other mental health problems. Your doctor may need to prescribe a different medicine, change your dose of other medicines, or change your dose of CERDELGA. Tell your doctor about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements before you start taking them.
Before taking CERDELGA, tell your doctor about all of your medical conditions, including heart problems (including a condition called long QT syndrome), a history of heart attack, kidney or liver problems. If you are pregnant or plan to become pregnant or breastfeed, talk to your doctor. It is not known if CERDELGA will harm your unborn baby. Talk to your doctor if you are breastfeeding or planning to breastfeed. It is not known if CERDELGA passes into your breast milk. You and your doctor will decide if you should take CERDELGA or breastfeed. You should not do both.
CERDELGA, used with certain other medicines, may cause changes in the electrical activity of your heart (ECG changes) and irregular heart beat (arrhythmias). Tell your doctor if you have new symptoms such as palpitations, fainting, or dizziness.
The most common side effects (≥10%) of CERDELGA include: tiredness, headache, nausea, diarrhea, back pain, pain in extremities, and upper abdominal pain. Call your doctor for medical advice about adverse effects.
Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of CERDELGA. Call your doctor for medical advice about side effects.
It is not known if CERDELGA is safe and effective in children.
Please see the full Prescribing Information, including the Patient Medication Guide, for CERDELGA.