CareConnect Personalized Support Services
Navigating life with a rare disease? We want you to know you aren’t alone. You have an experienced team ready to provide you with individualized support.
CareConnect Personalized Support Services is a Sanofi-sponsored support program for people with lysosomal storage disorders (LSDs) and their families. We offer personalized disease education, information about treatment options, and resources that can help you with access to treatment.
Meet your dedicated team
Case Manager
Case Managers have deep experience navigating the healthcare system and helping families manage health insurance concerns.
They can help you understand how to handle out-of-pocket costs or they can help you understand your options if you are without insurance.
Patient Education Liaison
Patient Education Liaisons (PELs) have clinical backgrounds in nursing or genetic counseling.
They can provide in-person disease and treatment education. They can also help you understand how the disease may be passed on in families.
Our range of support to help patients living with a rare disease includes:
Resources
Find educational resources to help you learn more about your GD1 and treatment.
Access
Learn how to access treatment
and understand insurance
options and available financial
assistance.
Coordination
Work with our experienced
team to assist you with life
transitions that impact access
to treatment or insurance.
Community
Join a supportive community of individuals who live with GD1.
CareConnect represents Sanofi’s 40-year commitment to supporting the rare disease community
The CareConnect program is free, voluntary, and confidential. To enroll, simply click on the button below. You can complete your enrollment form and submit online in minutes. You can also contact our team directly at 1-800-745-4447, option 3, or by email (info@CareConnectPSS.com) with any questions.
FINANCIAL ASSISTANCE
If affordability is an obstacle, CareConnect may be able to help
CareConnect offers programs that may be able to help eligible patients manage out-of-pocket costs related to their therapy. If you or your loved one cannot access treatment through a health plan or do not have healthcare coverage, we may be able to help.
CareConnect Copay Program*
The CareConnect Copay Program helps eligible patients in the U.S. who are prescribed certain Sanofi therapies pay for eligible out-of- pocket drug costs and specified infusion-related charges, including copays, coinsurance, and deductibles, up to the program maximum.
CareConnect Patient Assistance Program
The CareConnect Patient Assistance Program was established in the U.S. to provide certain Sanofi therapies at no cost to eligible patients who do not have health insurance or cannot access an eligible product under the terms of their insurance plan. This program is considered a temporary solution until coverage can be secured.
Additional Assistance Programs
CareConnect offers additional programs designed to solve obstacles encountered while accessing and maintaining therapy.
*Not valid for prescriptions paid, in whole or in part, by Medicaid, Medicare, VA, DOD, TRICARE, or other federal or state programs including any state pharmaceutical assistance programs. No claim for reimbursement of any out-of-pocket costs covered by the Copay Program may be submitted to any third-party payer, whether public or private, including but not limited to patients’ insurance, Flexible Spending Account (FSA), or Health Savings Account (HSA), or any other type of medical savings account. This program is not valid where prohibited by law, taxed, or restricted. Sanofi reserves the right to rescind, revoke, terminate, or amend this offer, eligibility, and terms of use at any time without notice. Any savings provided by the program may vary depending on patients’ out-of-pocket costs. Patients will receive all program details upon registration.
PATIENT CHECKLIST
This easy-to-follow checklist can help you prepare to start your GD1 treatment on Cerdelga. It also has important reminders once your doctor has prescribed Cerdelga.
Download Cerdelga Patient Checklist
DOCTOR DISCUSSION GUIDE
This guide includes a quick look at data from Cerdelga’s clinical trials. Use this discussion guide to help you remember what you would like to talk about with your doctor.
Download Doctor Discussion Guide
FAQs
Here are answers to some of the most frequently asked questions about Gaucher disease type 1 (GD1) and Cerdelga.
Sanofi does not provide medical advice, diagnosis, or treatment. The health information contained herein is provided for general educational purposes only. 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.
- Low hemoglobin levels (anemia)
- Low platelet counts (thrombocytopenia)
- Enlarged liver and/or spleen (the liver and the spleen can swell to multiple times their normal size)
- Bone disease (bone pain, loss of bone mineral density, and bone marrow changes)
- St. John’s Wort (Hypericum perforatum)
- Fungal infections
- Tuberculosis
- Seizures
- Heart conditions or high blood pressure
- Depression or other mental health problems
- Have heart problems, including a condition called long QT syndrome
- Have a history of heart attack
- Have kidney or liver problems
- Are pregnant or planning to become pregnant. It is not known if Cerdelga will harm your unborn baby
- Are breastfeeding or plan 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
Gaucher (pronounced go-SHAY) disease type 1 is a rare disorder affecting approximately 1 in 40,000 people in the general population. While it affects people from all ethnic groups, it is more prevalent in people of Ashkenazi Jewish heritage.
Gaucher disease type 1 (GD1) is an inherited disorder. This means the changes within the gene that cause the disorder were most likely passed down to you from your birth parents. There are many types of genetic disorders. GD1 is called an autosomal recessive disorder. Learn more about Gaucher inheritance.
Common signs and symptoms of Gaucher disease type 1 (GD1) include:
Gaucher disease type 1 (GD1) is a chronic, lifelong condition. While there is no cure, there are treatment options to help manage the signs and symptoms of GD1.
Cerdelga is a prescription medicine used for the long-term treatment of Gaucher disease type 1 (GD1) in adults. Cerdelga is not used in certain people with GD1. Your doctor will perform a test to make sure that Cerdelga is right for you. It is not known if Cerdelga is safe and effective in children.
People with Gaucher disease type 1 (GD1) have reduced activity of the enzyme that normally breaks down glucosylceramide (GL-1), causing GL-1 to build up. GL-1 buildup causes the signs and symptoms of GD1.
Cerdelga is a substrate reduction therapy (SRT) that treats GD1 by partially reducing the body’s ability to create GL-1, reducing the buildup of GL-1 in target organs, allowing the body’s remaining active enzyme to break down GL-1.
Watch a video on how Cerdelga works.
Cerdelga is used in certain, but not all, adults with Gaucher disease type 1 (GD1). Before prescribing Cerdelga, your doctor should perform a blood test to determine your CYP2D6 metabolizer status. This test helps decide if Cerdelga is right for you. Cerdelga can only be used by people with the following metabolizer rates: extensive metabolizers (EMs), intermediate metabolizers (IMs), and poor metabolizers (PMs). Your doctor will also consider other factors before prescribing Cerdelga.
People with certain metabolizer rates: Cerdelga may not work as well for people who have greater-than-usual CYP2D6 function, called ultra-rapid metabolizers (URMs). When the rate at which certain people’s bodies break down Cerdelga cannot be determined, this is called indeterminate metabolizer status. For these people, a certain dose of Cerdelga cannot be recommended.
Certain patients should not use Cerdelga based on their CYP2D6 metabolizer status due to an increased risk of side effects, including heart problems. Some patients with kidney disease and/or liver disease should avoid using Cerdelga, depending on their CYP2D6 metabolizer status.
Cerdelga can affect the way other medicines work, and other medicines can affect how Cerdelga works. Other medicines can impact the amount of Cerdelga in your blood. This depends on your metabolizer status and how the other medications are metabolized. In some cases, Cerdelga should not be used and in other cases the dose should be adjusted. Therefore, it is important to tell your healthcare providers about any prescription medicines, over-the-counter drugs, vitamins, supplements, or herbal remedies you are taking.
Especially tell your doctor if you take:
Medicine for:
Tell your doctor about any new medicines before you start taking them.
People with certain health conditions: Before taking Cerdelga, tell your doctor about all other medical conditions, including if you:
Ready to start Cerdelga?
Most adult patients with Gaucher disease type 1 are eligible for Cerdelga.
Want to know more about how Cerdelga works?
This video explains how Cerdelga works in your body to treat Gaucher disease type 1.
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.