公开(公告)号：US20230381287A1

公开(公告)日：2023-11-30

申请号：US18231982

申请日：2023-08-09

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Yung Hee Park ,  Nancy Chen ,  Jun Hu ,  Muthuraman Meiyappan ,  Thomas Alllen Miller

IPC: A61K38/47 ,  A61K31/445 ,  A61K47/02 ,  A61K47/12 ,  A61K47/26

CPC classification number: A61K38/47 ,  A61K31/445 ,  A61K47/02 ,  A61K47/12 ,  A61K47/26 ,  A61K9/0019

Abstract: The invention provides a composition of glucocerebrosidase, such as velaglucerase alfa, and isofagomine, in a molar ratio of at least about 1:2.5. Also provided is a use of the composition for treatment of a disorder related to a dysfunction in a GCase pathway. The disorder could be a lysosomal storage disease, such as Gaucher disease, Fabry disease, Pompe disease, a mucopolysaccharidoses, or multiple system atrophy. The disorder could also be a neurodegenerative disorder, such as Parkinson disease, Alzheimer's disease, or Lewy body dementia. The composition can have 0.5 to 5.0 mg/kg of glucocerebrosidase and isofagomine in at least about a 3-fold molar excess to the glucocerebrosidase. The composition can be administered intravenously or subcutaneously.