Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

February 2022 | 71 pages | ID: L7BEE71216A4EN
Global Markets Direct

Abstracts

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

SUMMARY

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Lysosomal alpha-glucosidase is an enzyme encoded by the GAA gene. It is essential for the degradation of glycogen to glucose in lysosomes. Defects in this gene lead to glycogen storage disease II or Pompe disease.

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 20 molecules. Out of which approximately 17 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase II, Preclinical and Discovery stages are 2, 3, 10 and 2 respectively. Similarly, the universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.

The latest report Lysosomal Alpha Glucosidase - Drugs In Development, 2022, outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. It also reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects.

The report is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

SCOPE

• The report provides a snapshot of the global therapeutic landscape for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
• The report reviews Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
• The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
• The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
• The report reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics and enlists all their major and minor projects
• The report assesses Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
• The report summarizes all the dormant and discontinued pipeline projects
• The report reviews latest news and deals related to Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics

REASONS TO BUY

• Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
• Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
• Identify and understand the targeted therapy areas and indications for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
• Identify the use of drugs for target identification and drug repurposing
• Identify potential new clients or partners in the target demographic
• Develop strategic initiatives by understanding the focus areas of leading companies
• Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
• Devise corrective measures for pipeline projects by understanding Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) development landscape
• Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

Contents

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
Denali Therapeutics Inc
Equaly SA
Genzyme Corp
ImmunityBio Inc
JCR Pharmaceuticals Co Ltd
M6P Therapeutics
NanoMedSyn 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
Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
History of Events
Recombinant 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 Canada for patients with late-onset Pompe disease (acid a-glucosidase deficiency)
Nov 12, 2021: Sanofi announces results of CHMP re-examination 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 International Annual Congress of the World Muscle Society
Sep 17, 2021: CHMP announces re-examinations 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 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

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