Medications for Fabry Disease

Storage disease due to deficiency of ceramide trihexosidase.

Drugs used to treat Fabry Disease

The following list of medications are in some way related to, or used in the treatment of this condition.

Drug name	Rating	Reviews	Rx/OTC	Pregnancy	CSA
Fabrazyme	Rate	Add review	Rx	B	N
Generic name: agalsidase beta systemic Drug class: lysosomal enzymes For consumers: dosage, interactions, side effects For professionals: Prescribing Information

Galafold	Rate	Add review	Rx		N
Generic name: migalastat systemic Drug class: miscellaneous metabolic agents For consumers: dosage, interactions, side effects

agalsidase beta	Rate	Add review	Rx	B	N
Generic name: agalsidase beta systemic Brand name: Fabrazyme Drug class: lysosomal enzymes For consumers: dosage, interactions, side effects For professionals: A-Z Drug Facts, AHFS DI Monograph

migalastat	Rate	Add review	Rx		N
Generic name: migalastat systemic Brand name: Galafold Drug class: miscellaneous metabolic agents For consumers: dosage, interactions, side effects For professionals: A-Z Drug Facts, AHFS DI Monograph

Rating	For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity	Activity is based on recent site visitor activity relative to other medications in the list.
Rx	Prescription Only.
OTC	Over the Counter.
Rx/OTC	Prescription or Over the Counter.
Off-label	This medication may not be approved by the FDA for the treatment of this condition.
EUA	An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.

Pregnancy Category
A	Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B	Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C	Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D	There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X	Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N	FDA has not classified the drug.

Controlled Substances Act (CSA) Schedule
M	The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U	CSA Schedule is unknown.
N	Is not subject to the Controlled Substances Act.
1	Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2	Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3	Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4	Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5	Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.

Alcohol
X	Interacts with Alcohol.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.