JPWO2021178737A5

JPWO2021178737A5 -

Info

Publication number: JPWO2021178737A5
Application number: JP2022553583A
Authority: JP
Publication date: 2024-03-14

Claims

A medicament for the treatment of Fabry disease in a human patient in need of treatment, comprising a therapeutically effective dose of migalastat or a salt thereof, wherein the patient is: A29D, R38S, N53Y, Y88C, V124G. , I133F, A143V, Y152N, F159C, A160D, D165N, F169I, L180V, D182G, R196T, W209R, A257T, P259S, G271A, S276T, M290V, A291S, I303T, I303V, L310V, G3 60A, G360R, G375A, L394P, G411S and N419D .

(i) said migalastat or a salt thereof is administered to said patient every other day ;
(ii) said patient is administered about 100 to about 150 mg free base equivalent of said migastat or a salt thereof every other day;
(iii) said patient is administered about 123 mg free base equivalent of said migalastat or a salt thereof every other day;
(iv) said patient is administered about 123 mg of migalastat free base every other day; or
(v) said patient is administered about 150 mg of migalastat hydrochloride every other day;
The medicament according to claim 1.

The medicament according to claim 1 or 2, wherein the migastat or a salt thereof is administered orally or by injection .

The medicament according to any one of claims 1 to 3 , wherein the migalastat or a salt thereof enhances α-galactosidase A activity.

The medicament according to any one of claims 1 to 4 , wherein the patient is male or female .

Medicament according to any one of claims 1 to 5 , wherein said mutations are disclosed in a pharmacological reference table.

7. The pharmacological reference table of claim 6 , wherein the pharmacological lookup table is provided in a package insert for a migastat product approved to treat Fabry disease, a package insert for GALAFOLD®, or a website. medicine .

The pharmacological reference table is provided on a website , and the website is available at www. galafoldamenabilitytable. com or www. fabrygenevariantsearch. 7. The medicament according to claim 6 , wherein the medicament is one or more of com.

A medicament for enhancing alpha-galactosidase A in a patient diagnosed with Fabry disease or suspected of having Fabry disease , comprising a therapeutically effective dose of migalastat or a salt thereof , wherein the patient has: A29D , R38S, N53Y, Y88C, V124G, I133F, A143V, Y152N, F159C, A160D, D165N, F169I, L180V, D182G, R196T, W209R, A257T, P259S, G271A, S276T, M290V, A291S , I303T, I303V, L310V, G360A , G360R, G375A, L394P, G411S and N419D .

(i) said migalastat or a salt thereof is administered to said patient every other day ;
(ii) said patient is administered about 100 to about 150 mg free base equivalent of said migastat or a salt thereof every other day;
(iii) said patient is administered about 123 mg free base equivalent of said migalastat or a salt thereof every other day;
(iv) said patient is administered about 123 mg of migalastat free base every other day; or
(v) said patient is administered about 150 mg of migalastat hydrochloride every other day;
The medicament according to claim 9 .

The medicament according to claim 9 or 10 , wherein the migastat or a salt thereof is administered orally or by injection .

The medicament according to any one of claims 9 to 11 , wherein the patient is male or female .

Medicament according to any one of claims 9 to 12 , wherein said mutations are disclosed in a pharmacological reference table.

14. The pharmacological lookup table is provided in a package insert for a migastat product approved to treat Fabry disease , in a package insert for GALAFOLD®, or on a website. medicine .

The pharmacological reference table is provided on a website , and the website is available at www. galafoldamenabilitytable. com or www. fabrygenevariantsearch. 14. The medicament according to claim 13 , wherein the medicament is one or more of com.