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Request more information or a visit from your Amicus Rare Disease Specialist

Find out if Galafold® (migalastat) may be an appropriate treatment for your adult patients with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant. Once you submit your request, a representative will contact you.

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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.

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.

Identify amenable GLA variants

Rebecca, living with Fabry disease

In the search bars below, enter up to 3 GLA variants to determine amenability.

You can use this search tool to find out whether a specific GLA variant has been listed in the Full Prescribing Information as amenable to treatment with Galafold. The current list of amenable GLA variants can also be found in the Full Prescribing Information.

Whether a certain amenable GLA variant in a patient with Fabry disease is disease-causing or not should be determined by the prescribing physician (in consultation with a clinical genetics professional, if needed) prior to treatment initiation. Consultation with a clinical genetics professional is strongly recommended in cases where the amenable GLA variant is of uncertain clinical significance (VUS, variant of uncertain significance) or may be benign (not causing Fabry disease).

You can use this search tool to find out whether a specific GLA variant has been listed in the Full Prescribing Information as amenable to treatment with Galafold. The current list of amenable GLA variants can also be found in the Full Prescribing Information.

Thank you! Your submission has been received!
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This search bar is intended only for Healthcare Professionals licensed to practice in the United States.

For DNA Change

Please use format c.#A>B or c.A#B for DNA changes, where 'c.' is optional; # indicates a number; A and B are letters. Examples: c.8T>C or c.T8C

For Protein Change

Protein changes can be written as either a single-letter or three-letter code. For example, p.(L3P) can be written as is or written as p.(Leu3Pro), where the ‘p.’ and ‘()’ are optional in both cases.

Whether a certain amenable GLA variant in a patient with Fabry disease is disease-causing or not should be determined by the prescribing physician (in consultation with a clinical genetics professional, if needed) prior to treatment initiation. Consultation with a clinical genetics professional is strongly recommended in cases where the amenable GLA variant is of uncertain clinical significance (VUS, variant of uncertain significance) or may be benign (not causing Fabry disease).

*
Note: This is only for searching multiple GLA variants on the same chromosome.

You can use this search tool to find out whether a specific GLA variant has been listed in the Full Prescribing Information as amenable to treatment with Galafold. The current list of amenable GLA variants can also be found in the Full Prescribing Information.

Thank you! Your submission has been received!
Oops! Something went wrong while submitting the form.

This search bar is intended only for Healthcare Professionals licensed to practice in the United States.

For DNA Change

Please use format c.#A>B or c.A#B for DNA changes, where 'c.' is optional; # indicates a number; A and B are letters. Examples: c.8T>C or c.T8C

For Protein Change

Protein changes can be written as either a single-letter or three-letter code. For example, p.(L3P) can be written as is or written as p.(Leu3Pro), where the ‘p.’ and ‘()’ are optional in both cases.

Whether a certain amenable GLA variant in a patient with Fabry disease is disease-causing or not should be determined by the prescribing physician (in consultation with a clinical genetics professional, if needed) prior to treatment initiation. Consultation with a clinical genetics professional is strongly recommended in cases where the amenable GLA variant is of uncertain clinical significance (VUS, variant of uncertain significance) or may be benign (not causing Fabry disease).

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.

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.

You can also determine whether your patient's GLA variant is amenable by:

The Galafold Full Prescribing Information includes a list of GLA variants that have been identified as amenable to Galafold.

Download

For answers to questions about amenability, you can also contact Amicus Medical Information at
1-877-4-AMICUS or [email protected].

Contact Us

Help your patients determine if their GLA variant is amenable to Galafold

Learn more about Fabry
disease at FabryFacts.com

Understand more about the complexities of this disease, including signs, symptoms, how diagnosis can vary between genders, and much more.

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Download the Galafold
Patient Referral Form

This form serves a dual purpose: as a prescription for Galafold in some states and as a way for patients to register for AMICUS ASSIST®.

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