GALAFOLD- migalastat hydrochloride capsule

País: Estados Unidos

Língua: inglês

Origem: NLM (National Library of Medicine)

Ingredientes ativos:

MIGALASTAT HYDROCHLORIDE (UNII: CLY7M0XD20) (MIGALASTAT - UNII:C4XNY919FW)

Disponível em:

Amicus Therapeutics US, LLC

Via de administração:

ORAL

Tipo de prescrição:

PRESCRIPTION DRUG

Indicações terapêuticas:

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 [see Dosage and Administration (2.1) and Clinical Pharmacology (12.1)] . This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate [see Clinical Studies (14)] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. None. 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 background risk for major birth defects and miscarriage for the indicated population is unk

Resumo do produto:

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.

Status de autorização:

New Drug Application

Características técnicas

GALAFOLD- MIGALASTAT HYDROCHLORIDE CAPSULE
AMICUS THERAPEUTICS US, LLC
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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
RECENT MAJOR CHANGES
Dosage and Administration, Recommended Dosage and Administration (2.2)
6/2023
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. (1, 12.1)
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. (1)
DOSAGE AND ADMINISTRATION
Select adults with confirmed Fabry disease who have an amenable _GLA_
variant for treatment with
GALAFOLD. (2.1)
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). (2.1, 12.1)
The recommended dosage of GALAFOLD is 123 mg orally once every other
day. Take GALAFOLD at the
same time of day and do not take on consecutive days. Swallow capsule
whole. Do not cut, crush, or
chew the capsule. (2.2)
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. (2.2)
If the GALAFOLD dose is missed, take the m