국가: 미국
언어: 영어
출처: NLM (National Library of Medicine)
MIGALASTAT HYDROCHLORIDE (UNII: CLY7M0XD20) (MIGALASTAT - UNII:C4XNY919FW)
Amicus Therapeutics US, LLC
ORAL
PRESCRIPTION DRUG
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
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.
New Drug Application
GALAFOLD- MIGALASTAT HYDROCHLORIDE CAPSULE AMICUS THERAPEUTICS US, LLC ---------- 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 전체 문서 읽기