Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Overview
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Companies Involved in Therapeutics Development
Amicus Therapeutics Inc
Asklepios BioPharmaceutical Inc
Astellas Gene Therapies
AVROBIO Inc
Deli Therapeutics Inc
Equaly SA
Genzyme Corp
ImmunityBio Inc
JCR Pharmaceuticals Co Ltd
M6P Therapeutics
noMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles
(cipaglucosidase alfa + miglustat) - Drug Profile
Product Description
Mechanism Of Action
History of Events
ACTUS-101 - Drug Profile
Product Description
Mechanism Of Action
History of Events
AT-845 - Drug Profile
Product Description
Mechanism Of Action
History of Events
avalglucosidase alfa - Drug Profile
Product Description
Mechanism Of Action
History of Events
AVRRD-03 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Fusion Protein to Replace GAA for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
JR-162 - Drug Profile
Product Description
Mechanism Of Action
History of Events
M-021 - Drug Profile
Product Description
Mechanism Of Action
History of Events
M-023 - Drug Profile
Product Description
Mechanism Of Action
OXY-2810 - Drug Profile
Product Description
Mechanism Of Action
PGN-004 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Recombint Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Recombint Enzyme to Replace Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
RPV-002 - Drug Profile
Product Description
Mechanism Of Action
History of Events
SPK-3006 - Drug Profile
Product Description
Mechanism Of Action
History of Events
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Dormant Products
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Discontinued Products
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Product Development Milestones
Featured News & Press Releases
Feb 11, 2022: M6P Therapeutics presents promising preclinical data in Pompe disease at the 18th Annual WORLDSymposium 2022
Feb 08, 2022: Nexviazyme (avalglucosidase alfa) shows sustained improvements in respiratory function and mobility in patients with Pompe disease
Feb 07, 2022: Astellas announces positive safety data from the FORTIS study of AT845 in adults with late-onset Pompe disease
Dec 03, 2021: European Medicines Agency validates Amicus Therapeutics marketing authorization applications for AT-GAA for the treatment of Pompe disease
Nov 29, 2021: Sanofi’s Pompe Disease drug Nexviazyme now available in Japan
Nov 18, 2021: The Lancet Neurology publishes pivotal Phase 3 PROPEL study results of AT-GAA in late-onset Pompe disease
Nov 15, 2021: Nexviazyme (avalglucosidase alfa for injection) is now approved in Cada for patients with late-onset Pompe disease (acid a-glucosidase deficiency)
Nov 12, 2021: Sanofi announces results of CHMP re-examition of the New Active Substance status for avalglucosidase alfa, a potential new standard of care for the treatment of Pompe disease
Sep 29, 2021: U.S. FDA accepts filings for Amicus’ AT-GAA for the treatment of Pompe disease
Sep 27, 2021: Orsini Specialty Pharmacy selected By Sanofi as a limited distribution partner for Nexviazyme
Sep 20, 2021: Amicus Therapeutics announces presentations at the 26th Intertiol Annual Congress of the World Muscle Society
Sep 17, 2021: CHMP announces re-examitions of recommendations for Nexviadyme
Aug 09, 2021: FDA approves Sanofi’s Nexviazyme for Pompe disease treatment
Aug 06, 2021: FDA approves Nexviazyme (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease
Jul 27, 2021: Sanofi provides update on avalglucosidase alfa EU submission for patients with Pompe Disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
Number of Products under Development by Stage of Development, 2022
Number of Products by Mechanism of Actions, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Routes of Administration, 2022
Number of Products by Stage and Routes of Administration, 2022
Number of Products by Molecule Types, 2022
Number of Products by Stage and Molecule Types, 2022

List of Tables

List of Tables
Number of Products under Development by Stage of Development, 2022
Number of Products under Development by Therapy Areas, 2022
Number of Products under Development by Indication, 2022
Number of Products under Development by Companies, 2022
Products under Development by Companies, 2022
Products under Development by Companies, 2022 (Contd..1)
Number of Products under Investigation by Universities/Institutes, 2022
Products under Investigation by Universities/Institutes, 2022
Number of Products by Stage and Mechanism of Actions, 2022
Number of Products by Stage and Route of Administration, 2022
Number of Products by Stage and Molecule Type, 2022
Pipeline by Amicus Therapeutics Inc, 2022
Pipeline by Asklepios BioPharmaceutical Inc, 2022
Pipeline by Astellas Gene Therapies, 2022
Pipeline by AVROBIO Inc, 2022
Pipeline by Denali Therapeutics Inc, 2022
Pipeline by Equaly SA, 2022
Pipeline by Genzyme Corp, 2022
Pipeline by ImmunityBio Inc, 2022
Pipeline by JCR Pharmaceuticals Co Ltd, 2022
Pipeline by M6P Therapeutics, 2022
Pipeline by NanoMedSyn SAS, 2022
Pipeline by Oxyrane Belgium NV, 2022
Pipeline by Pharming Group NV, 2022
Pipeline by Spark Therapeutics Inc, 2022
Dormant Projects, 2022
Discontinued Products, 2022