Treatment of Fabry disease in ERT-naïve and ERT-experienced patients
Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-nave Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.
1. A method of reducing gastrointestinal symptoms in a Fabry patient exhibiting one or more gastrointestinal symptoms, the method comprising administering to the patient a formulation comprising an effective amount of migalastat or salt thereof every other day to thereby reduce one or more of the gastrointestinal symptoms, wherein the effective amount is about 123 mg free base equivalent (FBE), wherein the gastrointestinal symptoms comprise diarrhea, wherein the patient is an enzyme replacement therapy (ERT)-naïve patient and wherein the patient has a mutation in α-Galactosidase A selected from the group consisting of A156T, D33G, G144V, and M187I.
2. The method of claim 1 , wherein administration of migalastat or a salt thereof for at least 18 months to an enzyme replacement therapy (ERT)-naïve patient having diarrhea symptoms at baseline provides a decrease in diarrhea of greater than 1.0 as assessed using the Gastrointestinal-Symptoms-Rating-Scale (GSRS).
3. The method of claim 1 , wherein the patient is administered about 123 mg of migalastat free base every other day.
4. The method of claim 1 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.
5. The method of claim 1 , wherein the formulation comprises an oral dosage form.
6. The method of claim 5 , wherein the oral dosage form comprises a tablet, a capsule or a solution.
7. The method of claim 1 , wherein the migalastat or salt thereof is administered for at least 6 months.
8. The method of claim 1 , wherein the migalastat or salt thereof is administered for at least 18 months.
9. A method of reducing gastrointestinal symptoms in a Fabry patient exhibiting one or more gastrointestinal symptoms, the method comprising:
(i) identifying an enzyme replacement therapy (ERT)-naïve patient having Fabry disease and exhibiting diarrhea, reflux, and indigestion, and
(ii) administering to the patient migalastat or salt thereof every other day in an amount effective to reduce the diarrhea, reflux, and indigestion, wherein the effective amount is about 123 mg free base equivalent (FBE),
wherein the patient has a mutation in α-Galactosidase A selected from the group A156T, D33G, G144V, and M187I,
wherein the administration results in (a) a decrease in diarrhea of greater than 1.0 as assessed using the Gastrointestinal-Symptoms-Rating-Scale (GSRS), (b) a decrease in reflux of greater than 0.6 as assessed using the GSRS, and (c) a decrease in indigestion of greater than 0.5 as assessed using the GSRS.
10. The method of claim 9 , wherein the mutation is D33G.
11. The method of claim 9 , wherein the mutation is M187I.
12. The method of claim 1 , wherein the mutation is A156T.
13. The method of claim 1 , wherein the mutation is D33G.
14. The method of claim 1 , wherein the mutation is G144V.
15. The method of claim 1 , wherein the mutation is M187I.
16. The method of claim 9 , wherein the mutation is A156T.
17. The method of claim 9 , wherein the mutation is G144V.