Usual Adult Dose for Fabry Disease

123 mg orally once every other day

• Take at the same time of day on an empty stomach; do not consume food for at least 2 hours before and 2 hours after dosing; clear liquids may be consumed during this period

Comments:

• This drug should not be taken on 2 consecutive days.
• Patients with Fabry disease should have an amenable galactosidase alpha gene variant (GLA) that is interpreted by a clinical geneticist as pathogenic or likely pathogenic as causing Fabry disease.
• This drug approval is under an accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate; continued approval is subject to demonstrated clinical benefit.

Use: For the treatment of adults with Fabry disease and an amenable galactosidase alpha gene variant (GLA) based on in vitro assay data.

Renal Dose Adjustments

Mild or moderate renal impairment (eGFR 30 mL/min/1.73 or greater): No adjustment recommended
Severe renal impairment (eGFR less than 30 mL/min/1.72 m2): Not recommended

Liver Dose Adjustments

No adjustment recommended

Precautions

CONTRAINDICATIONS: None

Safety and efficacy have not been established in patients younger than 18 years.

Consult WARNINGS section for additional precautions.

Dialysis

Not recommended

Other Comments

Administration advice:

• Take 1 capsule once every other day at the same time of day
• Take on an empty stomach; do not consume food for 2 hours before and 2 hours after dosing; clear liquids may be consumed during this period.
• Swallow capsule whole; do not cut, crush, or chew

Missed Dose: If a dose is missed, take the missed dose only if it is within 12 hours of the normal time that dose should have been taken; if more than 12 hours have passed, resume taking at the next planned dosing day and time and according to the every-other day dosing schedule; do not take this drug on 2 consecutive days.

Storage requirements:

• Keep capsules in blister card until use; store between 68F and 77F (20C to 25C)

General:

• Consultation with a clinical geneticist is strongly recommended, especially in cases where the amenable galactosidase alpha gene (GLA) variant is of uncertain clinical significance (VUS [variant of uncertain significance] or may be benign (not causing Fabry disease).
• Tables of amenable GLA variants based on in vitro assay are available in the package labeling; further information may be obtained by contacting Amicus Medical Information at [email protected]

Patient advice:

• Read the US FDA-approved patient labeling (Instructions for Use).
• Patients should receive instructions on proper administration and what to do in the case of a missed dose.

Frequently asked questions

• How much does Galafold cost?