RESOURCES FOR ELIGIBLE PATIENTS
Sanofi CareConnectPSS® Program
Whether you’re newly diagnosed with Gaucher disease type 1 (GD1) or switching treatment, a member of the Sanofi CareConnectPSS team can be there to help you through each step. They offer a wide range of support services for you and your family that’s one-on-one, confidential, and personalized.
If you’re already a member of CareConnectPSS, you can keep the same dedicated Case Manager and Patient Education Liaison you’ve been working with closely. You can also explore other tools and resources that are available to you at CareConnectPSS.com.
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Understanding GD1
GD1 can be hard to understand or explain. CareConnectPSS offers disease and treatment education—both in person and virtually—for you, family members, and important members of your community.
Coordinating Care
It is important to stay connected to GD1 healthcare providers. CareConnectPSS can assist you in finding medical facilities. If you relocate or travel, CareConnectPSS can also help with logistics.
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Knowing Insurance Coverage
It is important to understand coverage. CareConnectPSS can help eligible patients navigate the healthcare system and know more about their options.
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Receiving Financial Assistance
CareConnectPSS can help eligible patients understand and manage treatment costs, follow up on insurance claims, and find assistance programs.
CareConnectPSS Co-Pay Assistance Program
- This program helps eligible US individuals, who are prescribed one of Sanofi’s treatments, pay for their eligible, out-of-pocket, drug-related expenses, including co-pays, coinsurance, and deductibles
- To be eligible, you or your child must:
- Be a resident of the United States
- Have commercial insurance
- Be prescribed one of Sanofi’s treatments*
- Have prescription drug coverage for your Sanofi treatment
- The program is not valid for prescriptions eligible to be reimbursed, in whole or in part, by Medicaid, Medicare (including Medicare Part D), or other federal or state programs (including any state prescription drug assistance programs). The program is also not valid where prohibited by law.
- Your CareConnectPSS Case Manager can provide more information about the Co-Pay Assistance Program and the application process, or you can call 1‑800‑745‑4447, Option 3
*The CareConnectPSS Co-Pay Program is available only in the United States and cannot be combined with any other rebate/coupon, free trial, or similar offer. Co-pay benefits are not transferable. This program assists patients with their out-of-pocket drug costs for their prescribed Sanofi treatment only and does not cover or provide support for the cost of MD office visits/evaluations, nursing services/observation periods, blood work, x-rays or other testing, premedications/other medications, transportation or other related services. No claim for reimbursement of any out-of-pocket expense covered by the CareConnectPSS Co-Pay Program may be submitted to any third-party payer, whether public or private. Sanofi reserves the right to make eligibility decisions, set program maximums, and rescind, revoke, or amend this program at any time without notice. Savings provided by the Co-Pay Program may vary depending on patients’ out-of-pocket costs. Program details provided upon registration.
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.
Getting Started
Complete the CareConnectPSS program application.
Helpful information to have on hand when you apply:
- Your treating physician's contact information
- Your health insurance card
Your eligibility is verified.
- Your application will be reviewed for eligibility. If you are eligible, you will be automatically enrolled in the program. Enrollment in the program is subject to confirmation.
Your healthcare provider or specialty pharmacy will also receive a phone call with instructions on how to submit claims for reimbursement through the program. Your enrollment in the program is effective from the date of approval through the end of the calendar year (calendar year is January 1 through December 31). If you are currently enrolled in the Co-Pay Assistance Program, you will automatically be enrolled in the program for the next calendar year (January 1 through December 31) unless you decide to opt out of the program or your insurance coverage changes and you are no longer eligible for the program.
DOCTOR DISCUSSION GUIDE
You will likely need treatment over the course of your lifetime to manage the symptoms of GD1. Your doctor can help you decide which treatment option is right for you. Use this discussion guide to help you remember what you would like to talk about with your doctor.
Download Doctor Discussion Guide
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
FAQs
Here are answers to some of the most frequently asked questions about 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)
- 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 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.
Cerdelga is a substrate reduction therapy (SRT). Instead of replacing the missing amount of enzyme, SRT reduces glucosylceramide (GL-1) production. In doing do, people with Gaucher disease type 1 (GD1) can use any available 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 GD1 are eligible for 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.