Fabrazyme®: Treating patients with Fabry disease for 20 years.
Fabrazyme helps patients with Fabry disease by replacing a missing or deficient enzyme. It has been used to treat people with Fabry disease for 20+ years and is backed by the safety and effectiveness shown in clinical studies.
FABRAZYME: FIRST FOR PATIENTS
We are deeply committed to supporting patients within the rare disease community and offer a comprehensive range of services geared toward education, access, and advocacy.
CareConnectPSS® is here to support your journey.
Sanofi acts first for patients. CareConnectPSS offers personalized support services, representing our more than 35-year commitment to supporting patients’ individual needs in the rare disease community.
OUR RANGE OF SUPPORT TO HELP PATIENTS LIVING WITH A RARE DISEASE INCLUDES:
Disease-specific information and resources
Care coordination for treatment
Your Patient Education Liaison can help educate you, your family, friends, teachers, or employers about Fabry disease.
Your Case Manager will guide you through every step of the journey. They can provide resources that may help you with out-of-pocket costs, as well as health insurance claims and billing.
Dedicated CareConnectPSS Case Managers and Patient Education Liaisons
Connect with us online:
Contact a Case Manager
1-800-745-4447, Option 3
FINANCIAL ASSISTANCE PROGRAMS
Designed to help eligible patients* living with a rare disease.
Copay Assistance Program
The CareConnectPSS Copay Assistance Program helps eligible patients in the United States who are prescribed Fabrazyme pay for their eligible out-of-pocket drug costs and certain infusion-related (mixing and administration of the drug as well as infusion supplies such as saline, IV tubing, etc.) expenses, including copays, coinsurance, and deductibles.
- To be eligible, you must meet certain conditions and must not have coverage or prescriptions paid for in part or full under any state or federally funded healthcare program
Once enrolled in CareConnectPSS, work with your HCP to learn more and apply for the Copay Assistance Program. View paper application here.
Patient Assistance Program
If you do not have insurance, or your insurance has denied coverage for Fabrazyme, you may be eligible to receive Fabrazyme through the CareConnectPSS Patient Assistance Program (PAP). To see if you qualify, you please complete a CareConnectPSS PAP application with your doctor.
*Patients whose medication or infusion-related costs are covered by a state or federal health care program, including but not limited to Medicare, Medicare Part D, Medigap, Medicaid, Veterans Affairs (VA), Department of Defense (DoD), or TRICARE, are not eligible for the Co-Pay Program. Patients must live in the US or a US territory. Patients must be eligible under applicable state law(s). Other terms and conditions of the Program apply. Co-Pay Program does not cover or provide support for MD office visits/evaluations, nursing services/observation periods, blood work, x-rays or other testing, pre-medications/other medications, transportation or other related services associated with treatment. In accordance with state law, infusion-related costs are not covered for commercially insured patients residing in MA or RI. Sanofi reserves the right to modify or discontinue the programs at any time without notice. Savings may vary depending on patients’ out-of-pocket costs. All program details provided upon registration.
REQUEST INFORMATION
Need more information?
Fill out our request information form to get more details on disease education, treatment information, financial support, and patient support.
Access to these and other services is voluntary, and patients are not obligated to begin treatment if they contact us. You and your physician will make all treatment-related decisions, and, most importantly, the privacy and security of your personal information is always protected.
FABRY REGISTRY
Committed to increasing the understanding of Fabry disease and improving the lives of those living with this rare condition.
The Fabry Registry, sponsored and administered by Sanofi, is the largest international patient registry database dedicated to Fabry disease. It is led by a group of doctors with extensive experience in managing patients with Fabry disease.
The Registry helps doctors and researchers understand how Fabry disease and Fabrazyme treatment affect different people.
Talk to your doctor about enrolling in the Registry, and please visit registrynxt.com for more information.
Support networks
There are a number of organizations dedicated to providing information and support to people living with Fabry disease and other genetic disorders. This listing is provided as a resource only and does not constitute an endorsement by Sanofi of any particular organization or its programming. Additional resources on this topic may be available and should be investigated. Sanofi does not review or control the content of non-Sanofi websites.
American Kidney Fund
A non-profit organization that provides comprehensive programs of kidney health awareness, education and prevention.
Fabry Disease Information
A Sanofi-sponsored website with extensive information on Fabry disease.
Fabry Support & Information Group (FSIG)
A nonprofit organization dedicated to raising awareness and providing support to you and caregivers of patients with Fabry disease.
Fabry Registry
Information for patients and practitioners on this Sanofi-sponsored patient registry.
Genetic Alliance
The world's leading nonprofit health advocacy organization committed to transforming health through genetics.
National Fabry Disease Foundation
The NFDF supports the Fabry community through education, assistance, identification, research, and advocacy.
National Kidney Fund
A nonprofit that is a lifeline for all people affected by kidney disease with an emphasis on enhancing lives through action, education and accelerating change.
National Organization for Rare Disorders (NORD)
NORD is a nonprofit federation of health organizations dedicated to helping people with rare "orphan" diseases and assisting the organization.
National Society of Genetic Counselors (NSGC)
An organization that will help you find a genetic counselor who is in your area or available by phone.
Glossary of terms
An enzyme that is missing, not working properly, or present in smaller-than-normal amounts in people with Fabry disease. It is normally found in the lysosomes.
A change in one of your genes.
A type of Fabry disease in which there is little or no functional alpha-GAL activity. Symptoms typically begin in childhood and adolescence.
A treatment for kidney failure that purifies your blood as a substitute for the normal function of your kidneys.
An electrocardiogram, which measures electrical activity of the heart.
An echocardiogram, which is an ultrasound of the heart.
Estimated glomerular filtration rate is a measure of kidney function and can be used to determine your stage of kidney disease.
A protein produced by the body that acts to chemically change other substances. Enzymes are involved in breaking down or chemically altering substances so that the body can use or get rid of them.
A genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A.
A piece of DNA that codes for a particular protein. Each gene occupies a specific location on a chromosome, which defines a person’s bodily makeup and function.
Affecting or affected by genes.
A change in one of your genes.
A type of fat (also known as GL-3) that accumulates in the blood vessel walls of people with Fabry disease as a result of a deficiency in alpha-galactosidase A.
An abbreviation for globotriaosylceramide.
The delivery of liquid medicine into the blood through a vein.
A condition in which the kidneys are damaged and can’t filter blood normally.
A small structure in most cells that acts as the digestive system of the cell. Lysosomes contain and make various enzymes that break down substances. In Fabry disease, GL-3 builds up in the lysosomes.
Also referred to as late-onset or atypical Fabry disease, is a type of Fabry disease in which symptoms generally begin later and may be limited to a single organ.
A type of study in which individuals are observed or certain outcomes are measured. No attempt is made to affect the outcome (eg, no treatment is given).
A type of study in which both the patients and the researchers are aware of the drug or treatment being given.
An inactive substance, which has no effect on your body, that is used in clinical trials for comparison to help researchers understand clinical study results.
An inactive substance, which has no effect on your body, that is used in clinical trials for comparison to help researchers understand clinical study results.
An inactive substance, which has no effect on your body, that is used in clinical trials for comparison to help researchers understand clinical study results.
A type of study in which both the patients and the researchers are aware of the drug or treatment being given.
A type of study in which both the patients and the researchers are aware of the drug or treatment being given.
An inactive substance, which has no effect on your body, that is used in clinical trials for comparison to help researchers understand clinical study results.
The presence of GL-3 in the plasma.
A transient ischemic attack, sometimes called a “ministroke,” is caused by a clot or blockage to the brain. The symptoms usually last a short time, with the blockage dissolving by itself.
See the clinical evidence
First to demonstrate long-term, real-world efficacy and safety data.
IMPORTANT SAFETY INFORMATION
IMPORTANT SAFETY INFORMATION
Fabrazyme can cause serious side effects, including:
Severe Allergic (anaphylaxis) and Hypersensitivity Reactions
Approximately 1% of patients who have received Fabrazyme either during a clinical study or after Fabrazyme was approved have experienced anaphylactic (allergic) or severe hypersensitivity reactions during their infusion. Life-threatening severe anaphylactic (allergic) or severe hypersensitivity reactions have been seen in patients during Fabrazyme infusions.
- These reactions included: swelling of the face, mouth and throat, narrowing of breathing airways, low blood pressure, hives, difficulty swallowing, rash, trouble breathing, flushing, chest discomfort, itching and nasal congestion.
- People who have experienced these reactions have required treatment including heart/lung resuscitation, oxygen, fluids given through a vein, hospitalization, and treatment with inhaled drugs called beta-adrenergic agonists to help open the breathing airways, antihistamines, epinephrine (also known as adrenaline), and a medication given through the vein called a corticosteroid (or steroid) which helps to decrease the body’s allergic reaction by decreasing inflammation.
- If you experience a severe allergic or anaphylactic reaction, your healthcare professional will immediately stop the infusion of Fabrazyme and provide you the necessary emergency medical treatment. Because of the possibility that severe hypersensitivity reactions may occur, appropriate medical support should be available during your Fabrazyme infusion.
In the clinical studies, some patients developed IgE antibodies or a reaction to an allergy skin test specific to Fabrazyme. IgE antibodies are a specific kind of antibody that can sometimes be produced by the body’s immune system during an allergic reaction.
- Higher amounts of hypersensitivity reactions were seen in adult patients whose immune systems repeatedly made anti-Fabrazyme antibodies and in patients who had high antibody titers (units used to measure how much anti-drug antibody your immune system is making) compared to adult patients with negative antibody titers.
- Your doctor should consider testing for IgE antibodies if you experience suspected allergic reactions. Providing Fabrazyme to patients who have experienced severe or serious allergic reactions to Fabrazyme should only be done after carefully considering the risks and benefits of continuing the treatment, and only under the direct supervision of a qualified healthcare professional and with appropriate medical support readily available.
Infusion-Associated Reactions
In clinical studies with Fabrazyme, 59% of patients experienced infusion-associated reactions during Fabrazyme administration, some of which were severe. Infusion-associated reactions are defined as adverse reactions occurring on the same day as your infusion. During the clinical trials, infusion-associated reactions occurred more frequently in patients who were positive for anti-Fabrazyme antibodies than in patients who did not have anti-Fabrazyme antibodies.
- For patients who have had reactions to their infusions, it is recommended that they be given anti-fever and antihistamine medications right before their next infusions. Infusion-associated reactions have happened in some patients even after taking these medications before their infusions.
- If an infusion-associated reaction occurs, slowing the infusion rate, stopping the infusion for a short time and/or giving more anti-fever and antihistamine medications and or steroids may improve the symptoms.
- If severe infusion-associated reactions happen, your healthcare professional should consider stopping the Fabrazyme infusion right away and should provide medical care for your condition. Severe reactions are generally managed by giving antihistamine medications, corticosteroids, fluids through the vein, and/or oxygen when needed. Because severe infusion-associated reactions may happen, medical treatment should be readily available during your Fabrazyme infusion.
- People with advanced Fabry disease may have heart problems which could put them at a higher risk for severe complications from infusion-associated reactions. These patients should be watched closely during their infusion if the decision is made to give them Fabrazyme.
Common and Other Possible Side Effects: Common side effects reported in 20% or more of Fabrazyme treated patients in clinical studies compared to placebo were upper respiratory tract infection, chills, fever, headache, cough, burning and/or tingling sensation, fatigue, swelling in the legs, dizziness and rash.
Please see full Prescribing Information for Fabrazyme.
INDICATION
Fabrazyme® is used to treat adults and children 2 years of age and older with confirmed Fabry disease.
IMPORTANT SAFETY INFORMATION
Show moreIMPORTANT SAFETY INFORMATION
Fabrazyme can cause serious side effects, including:
Severe Allergic (anaphylaxis) and Hypersensitivity Reactions
Approximately 1% of patients who have received Fabrazyme either during a clinical study or after Fabrazyme was approved have experienced anaphylactic (allergic) or severe hypersensitivity reactions during their infusion. Life-threatening severe anaphylactic (allergic) or severe hypersensitivity reactions have been seen in patients during Fabrazyme infusions.
- These reactions included: swelling of the face, mouth and throat, narrowing of breathing airways, low blood pressure, hives, difficulty swallowing, rash, trouble breathing, flushing, chest discomfort, itching and nasal congestion.
- People who have experienced these reactions have required treatment including heart/lung resuscitation, oxygen, fluids given through a vein, hospitalization, and treatment with inhaled drugs called beta-adrenergic agonists to help open the breathing airways, antihistamines, epinephrine (also known as adrenaline), and a medication given through the vein called a corticosteroid (or steroid) which helps to decrease the body’s allergic reaction by decreasing inflammation.
- If you experience a severe allergic or anaphylactic reaction, your healthcare professional will immediately stop the infusion of Fabrazyme and provide you the necessary emergency medical treatment. Because of the possibility that severe hypersensitivity reactions may occur, appropriate medical support should be available during your Fabrazyme infusion.
In the clinical studies, some patients developed IgE antibodies or a reaction to an allergy skin test specific to Fabrazyme. IgE antibodies are a specific kind of antibody that can sometimes be produced by the body’s immune system during an allergic reaction.
- Higher amounts of hypersensitivity reactions were seen in adult patients whose immune systems repeatedly made anti-Fabrazyme antibodies and in patients who had high antibody titers (units used to measure how much anti-drug antibody your immune system is making) compared to adult patients with negative antibody titers.
- Your doctor should consider testing for IgE antibodies if you experience suspected allergic reactions. Providing Fabrazyme to patients who have experienced severe or serious allergic reactions to Fabrazyme should only be done after carefully considering the risks and benefits of continuing the treatment, and only under the direct supervision of a qualified healthcare professional and with appropriate medical support readily available.
Infusion-Associated Reactions
In clinical studies with Fabrazyme, 59% of patients experienced infusion-associated reactions during Fabrazyme administration, some of which were severe. Infusion-associated reactions are defined as adverse reactions occurring on the same day as your infusion. During the clinical trials, infusion-associated reactions occurred more frequently in patients who were positive for anti-Fabrazyme antibodies than in patients who did not have anti-Fabrazyme antibodies.
- For patients who have had reactions to their infusions, it is recommended that they be given anti-fever and antihistamine medications right before their next infusions. Infusion-associated reactions have happened in some patients even after taking these medications before their infusions.
- If an infusion-associated reaction occurs, slowing the infusion rate, stopping the infusion for a short time and/or giving more anti-fever and antihistamine medications and or steroids may improve the symptoms.
- If severe infusion-associated reactions happen, your healthcare professional should consider stopping the Fabrazyme infusion right away and should provide medical care for your condition. Severe reactions are generally managed by giving antihistamine medications, corticosteroids, fluids through the vein, and/or oxygen when needed. Because severe infusion-associated reactions may happen, medical treatment should be readily available during your Fabrazyme infusion.
- People with advanced Fabry disease may have heart problems which could put them at a higher risk for severe complications from infusion-associated reactions. These patients should be watched closely during their infusion if the decision is made to give them Fabrazyme.
Common and Other Possible Side Effects: Common side effects reported in 20% or more of Fabrazyme treated patients in clinical studies compared to placebo were upper respiratory tract infection, chills, fever, headache, cough, burning and/or tingling sensation, fatigue, swelling in the legs, dizziness and rash.
Please see full Prescribing Information for Fabrazyme.
INDICATION
Fabrazyme® is used to treat adults and children 2 years of age and older with confirmed Fabry disease.