GALAFOLD (migalastat hcl)

There are 0 contraindications.

There are 0 severe contraindications.

There are 1 moderate contraindications. Clinically significant contraindication, where the condition can be managed or treated before the drug may be given safely.

Moderate List
No disease contraindications

There are 2 severe adverse reactions.

More Frequent	Less Frequent
None.	None.

Rare/Very Rare
Hepatocellular damage Hypertension

There are 38 less severe adverse reactions.

More Frequent	Less Frequent
Fever Headache disorder Nausea Pharyngitis Urinary tract infection	Acute abdominal pain Back pain Constipation Cough Depression Diarrhea Dizziness Dyspepsia Dyspnea Epistaxis Fatigue Fecal urgency Hypoesthesia Muscle spasm Myalgia Pain Palpitations Paresthesia Proteinuria Pruritus of skin Skin rash Vertigo Weight gain Xerostomia

Rare/Very Rare
Anorexia Drowsy Erythema Hypercholesterolemia Memory impairment Ocular itching Polyuria Reduced visual acuity Vomiting

Risk Summary: There were three pregnant women with Fabry disease exposed to migalastat hydrochloride in clinical trials. As such, the available data are not sufficient to assess drug associated risks of major birth defects, miscarriage, or adverse maternal or fetal outcomes. In animal reproduction studies, no adverse developmental effects were observed.

The estimated background risk for major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S.

general population, the background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Animal Data: No adverse developmental effects were observed with oral administration of migalastat to pregnant rats and rabbits during organogenesis at doses up to 26 and 54 times, respectively, the recommended dose based on AUC. No effects on post-natal development were observed following oral administration of up to 500 mg/kg migalastat twice daily to pregnant rats (16 times the recommended dose based on AUC) during organogenesis and through lactation.

Risk Summary: There are no human data available on the presence of migalastat in human milk, the effects on the breastfed infant, or the effects on milk production. Migalastat is present in the milk of lactating rats. When a drug is present in animal milk, it is likely that the drug will be present in human milk.

The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for migalastat hydrochloride and any potential adverse effects on the breastfed child from migalastat hydrochloride or from the underlying maternal condition. Animal Data: Migalastat concentrations in milk from rats following oral administration of up to 500 mg/kg twice daily (approximately 16 times the recommended human dose based on AUC) was approximately 2.5 times higher than levels in the rat maternal plasma at 4 hours post-dose. The concentration of migalastat in plasma from pups was approximately 11 times lower than the maternal plasma concentrations at 1 hour post-dose.

Clinical trials of migalastat hydrochloride did not include a sufficient number of patients 65 years and older to determine whether they respond differently from younger patients.