Pompe Disease Pipeline Assessment (2022): In-depth Analysis of Clinical Trials, Latest FDA, EMA, and PMDA Approvals, Emerging Drugs, Growth Prospects, and Key Companies

Pompe Disease pipeline constitutes 15+ key companies continuously working towards developing 20+ Pompe Disease treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

Pompe Disease Overview
Pompe disease belongs to the 'lysosomal storage disorders' (LSDs) group of diseases. Within the cells, lysosomes are tiny compartments where all sorts of substances are recycled. By the action of digestive enzymes, the compounds are degraded. There are currently more than 50 different LSDs that are considered to be caused by the deficiency of one of these enzymes. One such enzyme is acid alpha-glucosidase (GAA) and is responsible for glycogen's lysosomal degradation. Glycogen accumulates inside the lysosomes due to a lack or dysfunction of GAA, which eventually leads to cellular failure,cellular damage, tissue damage, and ultimately organ dysfunction. The dysfunction of the organ in Pompe disease is primarily expressed by muscle weakness and muscle wasting.

"Pompe Disease Pipeline Insight, 2022" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Pompe Disease Market.

The Pompe Disease Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

Some of the key takeaways from the Pompe Disease Pipeline Report https://www.delveinsight.com/report-store/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

• Companies across the globe are diligently working toward developing novel Pompe Disease treatment therapies with a considerable amount of success over the years. Pompe Disease Key players such as - M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas Gene Therapies, Asklepios Biopharmaceutical, Spark Therapeutics, Amicus Therapeutics, and others, are developing therapies for the Pompe Disease treatment
• Pompe Disease Emerging therapies such as - M 021, GYS1 Knockdown Oligonucleotide, MZE 001, AT845, AAV2/8LSPhGAA, SPK-3006, Cipaglucosidase alfa, and others are expected to have a significant impact on the Pompe Disease market in the coming years.
• In January 2016, Amicus Therapeutics initiated an open-label, fixed-sequence, ascending-dose, first-in-human study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of intravenous infusions of ATB200 co-administered with oral AT2221 in adult subjects with Pompe Disease.
• In December 2018, Amicus Therapeutics initiated a Phase III double-blind randomized study to assess the efficacy and safety of intravenous ATB200 co-administered with Oral AT2221 in adult subjects with lateonset Pompe Disease compared with Alglucosidase Alfa/Placebo
• In February 2020, Amicus Therapeutics initiated an open-label study of the safety, pharmacokinetics, efficacy, pharmacodynamics, and immunogenicity of ATB200/AT2221 in pediatric subjects aged0 to < 18 years with Pompe Disease. The trial is currently recruiting participants and is expected to be completed by June 2026.
• In May 2022, Amicus Therapeutics announced that the U.S. FDA had extended the review period by 90 days for the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat, the two components of AT-GAA. The revised PDUFA action dates for miglustat and cipaglucosidase alfa are August 29, 2022 and October 29, 2022, respectively.

Pompe Disease Pipeline Therapeutics Assessment
• Pompe Disease Assessment by Product Type
• Pompe Disease By Stage and Product Type
• Pompe Disease Assessment by Route of Administration
• Pompe Disease By Stage and Route of Administration
• Pompe Disease Assessment by Molecule Type
• Pompe Disease by Stage and Molecule Type

DelveInsight's Pompe Disease Report covers around 20+ products under different phases of clinical development like
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Emerging Pompe Disease Drugs Under Different Phases of Clinical Development Include:
• M 021: M6P Therapeutics
• GYS1 Knockdown Oligonucleotide: Entrada Therapeutics
• MZE 001: Maze Therapeutics
• AT845: Astellas Gene Therapies
• AAV2/8L SPhGAA: Asklepios Biopharmaceutical
• SPK-3006: Spark Therapeutics
• Cipa glucosidase alfa: Amicus Therapeutics

Get a Free Sample PDF Report to know more about Pompe Disease Pipeline Assessment and Clinical trials-
https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Pompe Disease Pipeline Analysis:

The Pompe Disease pipeline report provides insights into
• The report provides detailed insights about companies that are developing therapies for the Pompe Disease treatment with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Pompe Disease Treatment.
• Pompe Disease key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Pompe Disease Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Pompe Disease market.
The report is built using data and information traced from the researcher's proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Further Pompe Disease product details are provided in the report. Download the Pompe Disease pipeline report to learn more about the emerging Pompe Disease therapies at:
https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Pompe Disease Pipeline Market Drivers
• Improving Healthcare Infrastructure
• Demand for New and Effective Drugs
• Increasing research and development in the gene therapy and enzyme replacement therapy areas

Pompe Disease Pipeline Market Barriers
• Lack Of Cost-Effective Pompe Disease Treatment

Scope of Pompe Disease Pipeline Drug Insight
• Coverage: Global
• Key Pompe Disease Companies: M6P Therapeutics, Entrada Therapeutics, Maze Therapeutics, Astellas Gene Therapies, Asklepios Biopharmaceutical, Spark Therapeutics, Amicus Therapeutics, and others
• Key Pompe Disease Therapies: M 021, GYS1 Knockdown Oligonucleotide, MZE 001, AT845, AAV2/8LSPhGAA, SPK-3006, Cipaglucosidase alfa, and others
• Pompe Disease Therapeutic Assessment: Pompe Disease current marketed and Pompe Disease emerging therapies
• Pompe Disease Market Dynamics: Pompe Disease market drivers and Pompe Disease market barriers

Request for Sample PDF Report for Pompe Disease Pipeline Assessment and clinical trials- https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

Table of Contents
1 Pompe Disease Report Introduction
2 Pompe Disease Executive Summary
3 Pompe Disease Overview
4 Pompe Disease- Analytical Perspective In-depth Commercial Assessment
5 Pompe Disease Pipeline Therapeutics
6 Pompe Disease Late Stage Products (Phase II/III)
7 Pompe Disease Mid Stage Products (Phase II)
8 Pompe Disease Early Stage Products (Phase I)
9 Pompe Disease Preclinical Stage Products
10 Pompe Disease Therapeutics Assessment
11 Pompe Disease Inactive Products
12 Company-University Collaborations (Licensing/Partnering) Analysis
13 Pompe Disease Key Companies
14 Pompe Disease Key Products
15 Pompe Disease Unmet Needs
16 Pompe Disease Market Drivers and Barriers
17 Pompe Disease Future Perspectives and Conclusion
18 Pompe Disease Analyst Views
19 Appendix
20 About DelveInsight

Download Sample PDF Report to know more about Pompe Disease drugs and therapies- https://www.delveinsight.com/sample-request/pompe-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=gpr

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