30/19. Migalastat- (GALAFOLD)-@- ( Aug- 2018)- To treat adults with fabry disease
Drug Name:30/19. Migalastat- (GALAFOLD)-@- ( Aug- 2018)- To treat adults with fabry disease
List Of Brands:
Indication Type Description:
Indication
Adverse Reaction
Contra-Indications
Dosages/ Overdosage Etc
Patient Information
Pregnancy and lactation
Indication:
U.S. FDA APPROVED DRUGS DURING 2018
Sr.No- 30
HIGHLIGHTS OF PRESCRIBING INFORMATION
These highlights do not include all the information needed to use GALAFOLD safely and effectively. See full prescribing information for GALAFOLD. GALAFOLD™ (migalastat) capsules, for oral use
Initial U.S. Approval: 2018
INDICATIONS AND USAGE
GALAFOLD™ is an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone 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.
Adverse Reaction:
ADVERSE REACTIONS-
Most common adverse drug reactions = 10% are: headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia
Contra-Indications:
CONTRAINDICATIONS- None
Dosages/ Overdosage Etc:
DOSAGE AND ADMINISTRATION-
• Select adults with confirmed Fabry disease who have an amenable GLA variant for treatment with GALAFOLD.
•Treatment is indicated for patients with an amenable GLA variant that is interpreted by a clinical genetics professional as causing Fabry disease (pathogenic, likely pathogenic) in the clinical context of the patient. 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)
• The recommended dosage regimen of GALAFOLD is 123 mg orally once every other day at the same time of day. • Take on an empty stomach. Do not consume food at least 2 hours before and 2 hours after taking GALAFOLD to give a minimum 4 hours fast.
• Do not take GALAFOLD on 2 consecutive days. • If a dose is missed entirely for the day, take the missed dose only if it is within 12 hours of the normal time that the dose should have been taken. If more than 12 hours have passed, resume taking GALAFOLD at the next planned dosing day and time and according to the every-other-day dosing schedule. • Swallow capsules whole; do not cut, crush, or chew.
DOSAGE FORMS AND STRENGTHS- Capsules: 123 mg migalastat.
Patient Information:
PATIENT COUNSELING INFORMATION-
Advise the patient to read the FDA-approved patient labeling (Patient Information and Instructions for Use).
Administration-: Advise the patient:
• To take GALAFOLD once every other day at the same time of day.
• Take GALAFOLD on an empty stomach. Do not consume food at least 2 hours before and 2 hours after taking GALAFOLD to give a minimum 4 hours fast. Clear liquids can be consumed during this 4-hour period.
• Not to take GALAFOLD on 2 consecutive days.
• If a dose is missed entirely for the day, take the missed dose only if it is within 12 hours of the normal time that the dose should have been taken. If more than 12 hours have passed, resume taking GALAFOLD at the next planned dosing day and time, according to the every-other-day dosing schedule.
• Swallow capsules whole. Do not cut, crush, or chew.
Manufactured for: Amicus Therapeutics U.S., Inc. 1 Cedar Brook Drive Cranbury, NJ 08512 GALAFOLD™ is a trademark of Amicus Therapeutics, Inc.
Pregnancy and lactation:
USE IN SPECIFIC POPULATIONS-
1.Pregnancy Risk Summary - There were three pregnant women with Fabry disease exposed to GALAFOLD 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 (see Data). 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.
2. 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 GALAFOLD and any potential adverse effects on the breastfed child from GALAFOLD or from the underlying maternal condition.
3. Females and Males of Reproductive Potential Infertility- The effects of GALAFOLD on fertility in humans have not been studied.
Transient and fully reversible infertility in male rats was associated with migalastat treatment at a systemic exposure (AUC) equivalent to the human exposure at the recommended dose. Complete reversibility was seen at 4 weeks after the termination of treatment. Migalastat did not affect fertility in female rats
4. Pediatric Use- The safety and effectiveness of GALAFOLD have not been established in pediatric patients
5 Geriatric Use- Clinical trials of GALAFOLD did not include a sufficient number of patients 65 years and older to determine whether they respond differently from younger patients.
6. Renal Impairment- Migalastat is substantially excreted by the kidneys. Systemic exposure was significantly increased in subjects with severe renal impairment (eGFR less than 30 mL/min/1.73 m2). GALAFOLD has not been studied in patients with Fabry disease who have an eGFR less than 30 mL/min/1.73 m2.
GALAFOLD is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis. No dosage adjustment is required in patients with mild to moderate renal impairment (eGFR at least 30 mL/min/1.73 m2 and above)
