Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
The present invention provides dosing regimens for administering pharmacological chaperones to a subject in need thereof. The dosing regimens can be used to treat disorders caused by improper protein misfolding, such as lysosomal storage disorders.
1. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of 1-deoxygalactonojirimycin or a salt thereof, wherein the therapeutically effective dose provides one or more of:
a. a mean maximum drug plasma concentration (C max ) bioequivalent to about 9 μM;
b. a mean time to maximum drug plasma concentration (T max ) bioequivalent to about 3 hours; or
c. a mean drug plasma half-life (t 1/2 ) bioequivalent to about 3 hours,
wherein the 1-deoxygalactonojirimycin or salt thereof is administered every other day.
2. The method of claim 1 , wherein the 1-deoxygalactonojirimycin or salt thereof is administered as an oral dosage form.
3. The method of claim 2 , wherein the oral dosage form comprises a tablet, a capsule or a solution.
4. The method of claim 1 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity.
5. The method of claim 1 , wherein the patient is male.
6. The method of claim 1 , wherein the patient is female.
7. The method of claim 1 , wherein the therapeutically effective dose provides a C max bioequivalent to about 9 μM.
8. The method of claim 1 , wherein the therapeutically effective dose provides a T max bioequivalent to about 3 hours.
9. The method of claim 1 , wherein the therapeutically effective dose provides a t 1/2 bioequivalent to about 3 hours.
10. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of 1-deoxygalactonojirimycin or a salt thereof, wherein the therapeutically effective dose provides one or more of:
a. a mean maximum drug plasma concentration (C max ) within 20% of 9 μM;
b. a mean time to maximum drug plasma concentration (T max ) within 20% of 3 hours; or
c. a mean drug plasma half-life (t 1/2 ) within 20% of 3 hours,
wherein the 1-deoxygalactonojirimycin or salt thereof is administered every other day.
11. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof is administered as an oral dosage form.
12. The method of claim 11 , wherein the oral dosage form comprises a tablet, a capsule or a solution.
13. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity.
14. The method of claim 10 , wherein the patient is male.
15. The method of claim 10 , wherein the patient is female.
16. The method of claim 10 , wherein the therapeutically effective dose provides a C max within 20% of 9 μM.
17. The method of claim 10 , wherein the therapeutically effective dose provides a T max within 20% of 3 hours.
18. The method of claim 10 , wherein the therapeutically effective dose provides a t 1/2 within 20% of 3 hours.