Overview
Fabry Disease is treated with 4 medications in our database, including Galafold, ELFABRIO, Zavesca, Fabrazyme. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Amicus, Chiesi, Actelion, Sanofi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Fabry Disease treatment below.
Also known as:
Clinical phenotype terms— hover any for plain English:
X-linked recessive
Carried on the X chromosome; typically affects males more than females
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsUniversity Hospital, Toulouse
Chiesi Italia
Amicus Therapeutics — PHASE3
Núcleo de Apoio à Investigação Clínica - FMUP
Yonsei University
Takeda — NA
General University Hospital, Prague — NA
Chiesi Farmaceutici S.p.A. — PHASE2, PHASE3
IRCCS Azienda Ospedaliero-Universitaria di Bologna — NA
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
4 availableELFABRIO
1 INDICATIONS AND USAGE ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease. ELFABRIO is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the t…
1 INDICATIONS AND USAGE ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease. ELFABRIO is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adults with confirmed Fabry disease. ( 1 )
Fabrazyme
1 INDICATIONS AND USAGE FABRAZYME ® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. FABRAZYME is a hydrolytic lysosomal neutral gl…
1 INDICATIONS AND USAGE FABRAZYME ® is indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. FABRAZYME is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adult and pediatric patients 2 years of age and older with confirmed Fabry disease. ( 1 )
Galafold
1 INDICATIONS AND USAGE GALAFOLD is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ( GLA ) variant based on in vitro assay d…
1 INDICATIONS AND USAGE GALAFOLD is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ( GLA ) variant based on in vitro assay data [see Dosage and Administration (2.1) and Clinical Pharmacology (12.1) ] . This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate [see Clinical Studies (14) ] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. GALAFOLD is an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene ( GLA ) variant based on in vitro assay data. ( 1 , 12.1 ) This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. ( 1 )
Zavesca
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
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Treatment Centers
8 centersAnn & Robert H. Lurie Children's Hospital of Chicago
📍 Chicago, Illinois
University of Minnesota
📍 Minneapolis, Minnesota
Emory University
📍 Atlanta, Georgia
IRCCS Azienda Ospedaliero-Universitaria di Bologna
📍 Bologna
👤 Tommaso Pippucci, Biologist
UPMC Children's Hospital of Pittsburgh
📍 Pittsburgh, Pennsylvania
Peking University First Hospital
📍 Beijing, Beijing Municipality
Peking Union Medical College Hospital
📍 Beijing, Beijing Municipality
Emory University School of Medicine
📍 Atlanta, Georgia
Financial Resources
10 resourcesHealthWell Foundation — Fabry disease
HealthWell Foundation
Fabry disease
Good Days — Fabry disease
Good Days
Fabry disease
Patient Services Inc — Fabry disease
Patient Services Inc
Fabry disease
The Assistance Fund — Fabry disease
The Assistance Fund
Fabry disease
Patient Advocate Foundation — Fabry disease
Patient Advocate Foundation
Fabry disease
NORD Patient Assistance — Fabry disease
NORD Patient Assistance
Fabry disease
PAN Foundation — Fabry disease
PAN Foundation
Fabry disease
5 travel grants are also available for Fabry disease patients — see Travel Grants below ↓
Travel Grants
5 grantsPAN Foundation - Fabry Disease Premium Assistance Fund
PAN Foundation
Applicants must be receiving treatment for Fabry disease in the United States or U.S. territories and have health insurance that covers their qualifying medication. Additionally, their household income must be at or below 500% of the Federal Poverty Level.
National Kidney Foundation - Prescription Discount and Assistance Resources
National Assistance Foundation
Fabry Assist
Fabry Support & Information Group (FSIG)
The Fabry Assist Program provides one-time or infrequent financial support for US residents with Fabry disease for needs like travel to meetings and medical bills. Eligibility requires verification of necessity from a medical provider and is intended to cover expenses not addressed by other assistance programs.
Community
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4 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Fabry disease
What is Fabry disease?
Fabry Disease is treated with 4 medications in our database, including Galafold, ELFABRIO, Zavesca, Fabrazyme. 2 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Amicus, Chiesi, Actelion, Sanofi. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Fabry Disease treatment below.
How is Fabry disease inherited?
Fabry disease follows a x-linked recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Fabry disease?
Yes — 20 recruiting clinical trials are currently listed for Fabry disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Fabry disease?
25 specialists and care centers treating Fabry disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Fabry disease?
4 FDA-approved treatments and 10 patient support programs are currently tracked on UniteRare for Fabry disease. See the treatments and support programs sections for copay assistance, eligibility, and contact details.