Loading
If this is the first time anyone has visited this substance, we may be fetching live data from TGA Product Information, PBS, and state framework sources — that can take up to a minute. Cached substances load in a second or two.
Migalastat, sold under the brand name Galafold, is a medication used for the treatment of Fabry disease, a rare genetic disorder. Migalastat is an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone. It was developed by Amicus Therapeutics.
Read the full article on WikipediaFabry disease Clinical criteria: Treatment Phase: Initial treatment Patient must have at least one of: (i) documented deficiency of alpha-galactosidase enzyme activity in blood, (ii) presence of genetic mutations known to result in deficiency of alpha-galactosidase enzyme activity, AND Patient must have a documented migalastat amenable galactosidase alpha (GLA) gene variant, AND Patient must have an estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m2, AND Patient must be male with Fabry-related renal disease confirmed by at least one of the following: (i) abnormal albuminuria of more than 20 mcg/min, as determined by 2 separate samples at least 24 hours apart, (ii) abnormal proteinuria of more than 150 mg/24 hours, (iii) albumin:creatinine ratio greater than upper limit of normal in 2 separate samples at least 24 hours apart, (iv) renal disease due to long-term accumulation of glycosphingolipids in the kidneys; OR Patient must be female with Fabry-related renal disease confirmed by at least one of the following: (i) proteinuria of more than 300 mg/24 hours with clinical evidence of progression, (ii) renal disease due to long-term accumulation of glycosphingolipids in the kidneys; OR Patient must have Fabry-related cardiac disease confirmed by at least one of the following: (i) left ventricular hypertrophy, as evidenced by cardiac magnetic resonance imaging (MRI) or echocardiogram data, in the absence of hypertension, (ii) significant life-threatening arrhythmia or conduction defect, (iii) late gadolinium enhancement or a low T1 on cardiac MRI; OR Patient must have Fabry-related either: (i) ischaemic disease, (ii) cerebrovascular disease as shown on objective testing with no other cause or risk factors identified; OR Patient must have Fabry-related uncontrolled chronic pain despite the use of recommended doses of appropriate analgesia and antiseizure medications for peripheral neuropathy; OR Patient must have significant Fabry-related gastrointestinal symptoms despite the use of the recommended doses of appropriate pharmacological therapies. Treatment criteria: Must be treated by a physician with expertise in the management of Fabry disease. Population criteria: Patient must be at least 12 years of age. If hypertension is present in patients relying their eligibility on Fabry-related cardiac disease, the prescriber must treat it optimally for at least 6 months prior to submitting the first PBS authority application. Confirmation of eligibility for treatment with diagnostic reports including the confirmed mutations must be documented in the patient's medical records. The authority application must be made in writing and must include: (1) details of the proposed prescription; and (2) a completed authority application form relevant to the indication and treatment phase (the latest version is located on the website specified in the Administrative Advice).
“Fabry disease is a genetic disorder caused by various mutations of the enzyme α-GalA, which is responsible for breaking down the sphingolipid globotriaosylceramide (Gb3), among other glycolipids and glycoproteins. Some of these mutations result in misfolding of α-GalA, which subsequently fails protein quality control in the endoplasmic reticulum and is decomposed. Lack of functional α-GalA leads to accumulation of Gb3 in blood vessels and other tissues, with a wide range of symptoms including kidney, heart, and skin problems.”
“3–5 hours (single dose)”
Working under the parallel aged-care framework? Aged-care equivalent →