Methods Of Treating Fabry Disease
Provided are methods for the treatment of Fabry disease in a patient, such as reducing the risk of composite clinical outcomes. Also provided are methods for assessing various symptoms of Fabry disease such as gastrointestinal symptoms. Also provided are methods of evaluating treatment therapies for Fabry disease.
1 . A method of reducing the risk of composite clinical outcomes (CCO) in a female patient having Fabry disease, the method comprising administering to the female patient a formulation comprising an effective amount of migalastat or salt thereof every other day for at least 18 months, wherein the effective amount is about 100 mg to about 150 mg free base equivalent (FBE).
2 . The method of claim 1 , wherein the CCO comprises renal events, cardiac events, cerebrovascular events and death.
3 . The method of claim 2 , wherein the renal events comprise one or more of: a decrease in eGFR CKD-EPI ≥15 mL/min/1.73 m 2 , with the decreased eGFR<90 mL/min/1.73 m 2 relative to baseline; or an increase in 24-hour urine protein≥33%, with elevated protein≥300 mg relative to baseline.
4 . The method of claim 2 , wherein the cardiac events comprise one or more of: myocardial infarction; unstable cardiac angina; new symptomatic arrhythmia requiring antiarrhythmic medication, direct current cardioversion, pacemaker, or defibrillator implantation; or congestive heart failure [New York Association Class III or IV].
5 . The method of claim 2 , wherein the cerebrovascular events comprise one or more of stroke or transient ischemic attack.
6 . The method of claim 1 , wherein the migalastat or salt thereof enhances α-galactosidase A activity.
7 . The method of claim 1 , wherein the female patient is administered about 123 mg FBE of the migalastat or salt thereof every other day.
8 . (canceled)
9 . The method of claim 1 , wherein the female patient is administered about 150 mg of migalastat hydrochloride every other day.
10 . The method of claim 1 , wherein the formulation comprises an oral dosage form.
11 . The method of claim 10 , wherein the oral dosage form comprises a tablet, a capsule or a solution.
12 . The method of claim 1 , wherein the migalastat or salt thereof is administered for at least 3 years.
13 . The method of claim 1 , wherein the migalastat or salt thereof is administered for at least 4 years.
14 . The method of claim 1 , wherein the CCO incidence rate for a group of female patients on migalastat therapy for 18 months is less than 1.0 per patient·year.
15 . The method of claim 1 , wherein the CCO incidence rate for a group of female patients on migalastat therapy for 18 months is less than 0.5 per patient·year.
16 - 17 . (canceled)
18 . The method of claim 1 , wherein the female patient is an enzyme replacement therapy (ERT)-naïve patient.
19 . The method of claim 1 , wherein the female patient is an ERT-experienced patient.
20 . The method of claim 1 , wherein the female patient has a HEK assay amenable mutation in α-galactosidase A.
21 . The method of claim 20 , wherein the mutation is disclosed in a pharmacological reference table.
22 . The method of claim 21 , wherein the pharmacological reference table is provided in one or more of a product label for a migalastat product approved for the treatment of Fabry disease, a product label for GALAFOLD® and a website.
23 - 24 . (canceled)
25 . The method of claim 24 , wherein the website is one or more of www.galafoldamenabilitytable.com or www.fabrygenevariantsearch.com.
26 - 57 . (canceled)