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

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Published: February 01, 2022 Report Code: GMDGDHC22011TDB-ST

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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.

The Lysosomal Alpha – Drugs in Development research report provides a comprehensive overview on the therapeutics under development for Lysosomal Alpha, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type.  The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.  Additionally, the report provides an overview of key players involved in therapeutic development for Lysosomal Alpha and features dormant and discontinued projects.

Key Therapy Areas in the Lysosomal Alpha Pipeline Drugs Market

The key therapy area in the Lysosomal Alpha pipeline drugs market is Metabolic Disorders.

Key MoA in the Lysosomal Alpha Pipeline Drugs Market

The key mechanisms of action in the Lysosomal Alpha pipeline drugs market are Lysosomal Alpha Glucosidase Replacement, and Lysosomal Alpha Glucosidase Activator.

Lysosomal Alpha Pipeline Drugs Market Analysis, by MoA

Lysosomal Alpha Pipeline Drugs Market Analysis, by MoA

To get more insights on key MoA, download a free sample report

Lysosomal Alpha Pipeline Drugs Market Segmentation by RoA

The key routes of administration in the Lysosomal Alpha pipeline drugs market are Subcutaneous, Intravenous, Oral, and Intramuscular.

 Lysosomal Alpha Pipeline Drugs Market Analysis, by RoA

Lysosomal Alpha Pipeline Drugs Market Analysis, by RoA

To get more insights on key RoA, download a free sample report

Key Molecule Types in the Lysosomal Alpha Pipeline Drugs Market

The key molecule types in the Lysosomal Alpha pipeline drugs market are Small Molecule, Recombinant Enzyme, Fusion Protein, Gene Modified Cell Therapy, and Gene Therapy among others.

 Lysosomal Alpha Pipeline Drugs Market, by Molecule Type

Lysosomal Alpha Pipeline Drugs Market, by Molecule Type

To get more insights on key molecule types, download a free sample report

Major Companies in the Lysosomal Alpha Pipeline Drugs Market

The major companies in the Lysosomal Alpha pipeline drugs market are Amicus Therapeutics Inc, Asklepios BioPharmaceutical Inc, Astellas Gene Therapies, AVROBIO Inc, Denali Therapeutics Inc, eleva GmbH, Equaly SA, Genzyme Corp, ImmunityBio Inc, and M6P Therapeutics among others.

 Lysosomal Alpha Pipeline Drugs Market, by Major Companies

Lysosomal Alpha Pipeline Drugs Market, by Major Companies

For more company insights, download a free sample report

Lysosomal Alpha Pipeline Drugs Market Overview

Key Therapy Areas Metabolic Disorders
Key Mechanisms of action Lysosomal Alpha Glucosidase Replacement, and Lysosomal Alpha Glucosidase Activator
Key Routes of Administration Subcutaneous, Intravenous, Oral, and Intramuscular
Key molecule types Small Molecule, Recombinant Enzyme, Fusion Protein, Gene Modified Cell Therapy, and Gene Therapy
Major companies Amicus Therapeutics Inc, Asklepios BioPharmaceutical Inc, Astellas Gene Therapies, AVROBIO Inc, Denali Therapeutics Inc, eleva GmbH, Equaly SA, Genzyme Corp, ImmunityBio Inc, and M6P Therapeutics

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Lysosomal Alpha
  • The pipeline guide reviews pipeline therapeutics for Lysosomal Alpha by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Lysosomal Alpha therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Lysosomal Alpha therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA), and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews the latest news related to pipeline therapeutics for Lysosomal Alpha

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

  • Recognize emerging players with a potentially strong product portfolio and create effective counter strategies to gain a competitive advantage.

  • Find and recognize significant and varied types of therapeutics under development for Lysosomal Alpha

  • Classify potential new clients or partners in the target demographic.

  • Develop tactical initiatives by understanding the focus areas of leading companies.

  • Plan mergers and acquisitions meritoriously by identifying key players and their most promising pipeline therapeutics.

  • Formulate corrective measures for pipeline projects by understanding Lysosomal Alpha pipeline depth and focus of Indication therapeutics.

  • 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.

  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from the pipeline.

Table of Contents

Table of Contents

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

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

Table

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

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

Frequently asked questions

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