US 12,458,629 B2
Method for treating nonsense mutation mediated duchenne muscular dystrophy in pediatric patients
Ronald Kong, River Edge, NJ (US); and Joseph William McIntosh, Wyckoff, NJ (US)
Assigned to PTC THERAPEUTICS, INC., South Plainfield, NJ (US)
Appl. No. 17/602,576
Filed by PTC THERAPEUTICS, INC., South Plainfield, NJ (US)
PCT Filed Apr. 9, 2020, PCT No. PCT/US2020/027382
§ 371(c)(1), (2) Date Oct. 8, 2021,
PCT Pub. No. WO2020/210432, PCT Pub. Date Oct. 15, 2020.
Claims priority of provisional application 62/831,931, filed on Apr. 10, 2019.
Prior Publication US 2022/0175739 A1, Jun. 9, 2022
Int. Cl. A61K 31/4245 (2006.01); A61P 21/00 (2006.01)
CPC A61K 31/4245 (2013.01) [A61P 21/00 (2018.01)] 10 Claims
 
1. A method for ameliorating or managing nonsense mutation mediated Duchenne muscular dystrophy (nmDMD) in a human pediatric patient having nmDMD comprising, orally administering to a patient having an age in a range between greater than or equal to six months of age and an age less than two years of age a therapeutically effective amount of ataluren, or a pharmaceutically acceptable salt thereof, based on patient weight to attain an average plasma concentration of the ataluren or pharmaceutically acceptable salt thereof in the patient in a range of at least about 1 μg/mL to about 20 μg/mL during a 24 hour time period; wherein the therapeutically effective amount of the ataluren or pharmaceutically acceptable salt thereof is: (a) 40 mg/kg/day administered as a morning dose of 10 mg/kg, a midday dose of 10 mg/kg, and, an evening dose of 20 mg/kg, or (a) 80 mg/kg/day administered as a morning dose of 20 mg/kg, a midday dose of 20 mg/kg, and, an evening dose of 40 mg/kg.