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Last Updated: September 21, 2021

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Claims for Patent: 9,987,263


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Summary for Patent: 9,987,263
Title:Methods for treatment of Fabry disease
Abstract: Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.
Inventor(s): Lockhart; David J. (Del Mar, CA), Castelli; Jeff (New Hope, PA)
Assignee: Amicus Therapeutics, Inc. (Cranbury, NJ)
Application Number:15/338,923
Patent Claims: 1. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient an oral dosage form comprising 150 mg of 1-deoxygalactonojirimycin or a salt thereof every other day.

2. The method of claim 1, wherein the oral dosage form comprises a tablet, a capsule or a solution.

3. The method of claim 1, wherein the oral dosage form comprises a capsule.

4. The method of claim 1, wherein the 1-deoxygalactonojirimycin or a salt thereof enhances .alpha.-galactosidase A activity.

5. The method of claim 1, wherein the patient has an .alpha.-galactosidase A mutation selected from the group consisting of T41I, A143T, A97V, M51K, L300P, G328A, P205T and N215S.

6. The method of claim 1, wherein the patient is male.

7. The method of claim 1, wherein the patient is female.

8. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient an oral dosage form comprising 150 mg of migalastat hydrochloride every other day.

9. The method of claim 8, wherein the oral dosage form comprises a tablet, a capsule or a solution.

10. The method of claim 8, wherein the oral dosage form comprises a capsule.

11. The method of claim 8, wherein the migalastat hydrochloride enhances .alpha.-galactosidase A activity.

12. The method of claim 8, wherein the patient has an .alpha.-galactosidase A mutation selected from the group consisting of T41I, A143T, A97V, M51K, L300P, G328A, P205T and N215S.

13. The method of claim 8, wherein the patient is male.

14. The method of claim 8, wherein the patient is female.

15. The method of claim 1, wherein the patient has an .alpha.-galactosidase A mutation selected from the group consisting of A156V, D244N, E59K, F113L, G144V, G183D, G328A, I91T, L166V, L32P, M284T, M296I, M296V, N263S, Q279E, R363C, R363H, N34S, T41I, M51K, A97V, R112H, A143T, P205T, Y207S, N215S, P259R, N263S, L300P, R301Q, E358A, P409A, S201F, P205T and F295C.

16. The method of claim 8, wherein the patient has an .alpha.-galactosidase A mutation selected from the group consisting of A156V, D244N, E59K, F113L, G144V, G183D, G328A, I91T, L166V, L32P, M284T, M296I, M296V, N263S, Q279E, R363C, R363H, N34S, T41I, M51K, A97V, R112H, A143T, P205T, Y207S, N215S, P259R, N263S, L300P, R301Q, E358A, P409A, S201F, P205T and F295C.

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Serving leading biopharmaceutical companies globally:

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