Brand Name
Galafold
Generic Name
Migalastat
View Brand Information FDA approval date: August 10, 2018
Form: Capsule
What is Galafold (Migalastat)?
GALAFOLD is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene variant based on in vitro assay data [see Dosage and Administration.
Approved To Treat
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Related Clinical Trials
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants
Summary: An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects With Fabry Disease and Amenable GLA Variants and Severe Renal Impairment or End-Stage Renal Disease Treated With Hemodialysis
Summary: An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Summary: This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding. The study will evaluate outcomes of pregnancy and/or breastfeeding in women and infants exposed to migalastat.
Related Latest Advances
Brand Information
Galafold (migalastat hydrochloride)
1INDICATIONS AND USAGE
GALAFOLD is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate
2DOSAGE FORMS AND STRENGTHS
Capsules: 123 mg of migalastat in a size “2” capsule with an opaque blue cap and opaque white body with “A1001” printed in black, containing white to pale brown powder.
3CONTRAINDICATIONS
None.
4DESCRIPTION
Migalastat, an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone, is a low molecular weight iminosugar and an analogue of the terminal galactose of globotriaosylceramide (GL-3).
Migalastat is present in the form of a hydrochloride salt in GALAFOLD. The chemical name for migalastat hydrochloride is (+)-(2
Migalastat hydrochloride is a white to almost white crystalline solid. It is freely soluble in aqueous media within the pH range of 1.2 to 7.5.
GALAFOLD (migalastat) capsules for oral administration contain 123 mg of migalastat (equivalent to 150 mg migalastat hydrochloride) as a white to pale brown powder and are supplied in a size “2” hard gelatin capsule with an opaque blue cap and an opaque white body imprinted with “A1001” in black ink. The inactive ingredients are magnesium stearate and pregelatinized starch. Capsule shells consist of gelatin, indigotine - FD&C Blue 2, and titanium dioxide. The black ink consists of black iron oxide, potassium hydroxide, and shellac.
5CLINICAL STUDIES
Study AT1001‑011 (referred to as Study 1; NCT00925301) included a 6‑month randomized, double‑blind, placebo‑controlled phase followed by a 6‑month open‑label treatment phase and a 12‑month open‑label extension phase. Patients received 123 mg GALAFOLD orally every other day taken without consuming food 2 hours before and 2 hours after each dose to give a minimum 4‑hour fast
A total of 67 patients with Fabry disease who were naïve to GALAFOLD and enzyme replacement therapy (ERT) or were previously treated with ERT (agalsidase beta or non‑U.S. approved agalsidase alfa) and had been off ERT for at least 6 months were randomized in a 1:1 ratio to receive either GALAFOLD 123 mg every other day or placebo for the first 6 months. In the second 6 months, all patients were treated with GALAFOLD.
Results - Patients with Fabry Disease with Amenable
Of the 67 enrolled patients, 50 patients (32 females, 18 males) had amenable
Efficacy was evaluated after 6 months of treatment in 45 of 50 patients with amenable
Results - Patients with Fabry Disease with Non‑Amenable
Of the 67 enrolled patients in Study 1, 17 patients had non‑amenable
6HOW SUPPLIED/STORAGE AND HANDLING
GALAFOLD capsules are supplied as 123 mg migalastat, size “2” capsules with an opaque blue cap and opaque white body filled with white to pale brown powder and imprinted with “A1001” in black ink.
GALAFOLD capsules are packaged as two 7‑count capsules blister strips with aluminum foil lidding encased in cardboard blister cards providing 14 capsules per wallet pack that supplies the drug product for 4 weeks (28 days).
Wallet pack containing 14 GALAFOLD capsules NDC 71904‑100‑01.
Store at USP Controlled Room Temperature of 20° to 25°C (68° to 77°F). Excursions are permitted between 15° to 30°C (59° to 86°F).
Store in the original packaging to protect from moisture.
7PATIENT COUNSELING INFORMATION
Advise the patient to read the FDA‑approved patient labeling (
Administration
Advise the patient:
- To take GALAFOLD once every other day at the same time of day and do not take on consecutive days
- Swallow capsule whole. Do not cut, crush, or chew the capsule
- Take GALAFOLD on an empty stomach. Do not consume food or caffeine at least 2 hours prior to and 2 hours after taking GALAFOLD to give a minimum 4 hour fast
- Water (plain, flavored, or sweetened), fruit juices without pulp, and caffeine‑free carbonated beverages can be consumed during the fasting period
- If the GALAFOLD dose is missed, take the missed dose if it is within 12 hours of the time that the dose should have been taken. If more than 12 hours have passed, take GALAFOLD at the next planned dosing day and time following the original every‑other‑day dosing schedule
- To inform the healthcare provider of all medicines the patient takes, including prescription and over‑the‑counter medicines, vitamins, and herbal supplements
Pregnancy and Lactation Study
Inform the patient and/or caregiver that there is a study that collects data on pregnant women with Fabry disease, and data on the effects of GALAFOLD on lactation in women with Fabry disease and their neonates and infants up to 1 year of age who are exposed through breast milk. Encourage the patient and/or caregiver to participate and state that participation is voluntary [see
Manufactured for:
GALAFOLD is a registered trademark of Amicus Therapeutics, Inc.
8PRINCIPAL DISPLAY PANEL - NDC: 71904-100-01 - Carton Label (Inner Sleeve)
9PRINCIPAL DISPLAY PANEL - NDC: 71904-100-01 - Carton Label (Outer Sleeve)
