Methods of Treating Fabry Disease in Pediatric Patients
Provided are methods for the treatment of Fabry disease in adolescent patient.
1 . A method of treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a formulation comprising therapeutically effective dose of migalastat or a salt thereof, wherein the patient is a pediatric patient.
2 . The method of claim 1 , wherein the patient has an age in a range of from about 2 year to about <18 year.
3 . The method of claim 1 , wherein the patient has a weight in a range of from about <15 kg to about ≥50 kg.
4 . The method of any one of claims 1-3 , wherein the therapeutically effective dose of migalastat or a salt thereof is in a range of from about 15 mg to about 150 mg every other day.
5 . The method of any one of claims 1-4 , wherein the therapeutically effective dose of migalastat hydrochloride at a dose in a range of from about 25 mg to about 150 mg every other day.
6 . The method of any one of claims 1-5 , wherein the therapeutically effective dose of migalastat FBE in a range of from about 15 mg to about 123 mg every other day.
7 . The method of claim 1 or 2 , wherein the patient has an age in a range of from about 12 year to about <18 year.
8 . The method of claim 7 , wherein the patient has a weight of about ≥25 kg.
9 . The method of claim 8 , wherein the therapeutically effective dose of migalastat hydrochloride is in a range of from about 80 mg to about 150 mg every other day.
10 . The method of claim 7 , wherein the patient has a weight of about >45 kg.
11 . The method of claim 10 , wherein the therapeutically effective dose of migalastat hydrochloride is about 150 mg every other day.
12 . The method of claim 10 or 11 , wherein the therapeutically effective dose of migalastat FBE is about 123 mg every other day.
13 . The method of claim 1 or 2 , wherein the patient has an age in a range of from 6 year to <12 year.
14 . The method of claim 13 , wherein the patient has a weight of about >25 kg.
15 . The method of claim 13 or 14 , wherein the therapeutically effective dose of migalastat hydrochloride is in a range of from about 80 mg to about 150 mg every other day.
16 . The method of claim 1 or 2 , wherein the patient has an age in a range of from 2 year to <6 year.
17 . The method of claim 16 , wherein the patient has a weight of about <35 kg.
18 . The method of claim 16 or 17 , wherein the therapeutically effective dose of migalastat hydrochloride is in a range of from about 40 mg to about 80 mg every other day.
19 . The method of any one of claims 1-18 , wherein the patient has an eGFR of about ≥60 mL/min/1.73 m 2 .
20 . The method of any one of claims 1-19 , wherein the migalastat or salt thereof enhances or prolongs α-galactosidase A activity.
21 . The method of any one of claims 1-20 , wherein the formulation comprises an oral dosage form.
22 . The method of claim 21 , wherein the oral dosage form comprises a tablet, a capsule or a solution.
23 . The method of any one of claims 1-22 , wherein the patient is male.
24 . The method of any one of claims 1-22 , wherein the patient is female.
25 . The method of any one of claims 1-24 , wherein the patient is an enzyme replacement therapy (ERT)-naïve patient.
26 . The method of any one of claims 1-25 , wherein the patient is an ERT-experienced patient who has stopped ERT for at least 14 days.
27 . The method of any one of claims 1-26 , wherein the patient has a HEK assay amenable mutation in α-galactosidase A.
28 . The method of claim 27 , wherein the mutation is disclosed in a pharmacological reference table.
29 . The method of claim 28 , wherein the pharmacological reference table is provided in a product label for a migalastat product approved for the treatment of Fabry disease.
30 . The method of claim 29 , wherein the pharmacological reference table is provided in a product label for GALAPOLD®.
31 . The method of claim 30 , wherein the pharmacological reference table is provided at a website.
32 . The method of claim 31 , wherein the website is one or more of www.galafoldamenabilitytable.com or www.fabrygenevariantsearch.com.