Pompe Disease Pipeline| 15+ Key Players & 20+ Pipeline Drugs | FDA, EMA, and PMDA Approvals, Clinical Trials

DelveInsight's, "Pompe Disease Pipeline Insights, 2022," report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in the Pompe Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

The Pompe Disease Pipeline is evolving with novel mechanisms of action (MoAs) that will bring positive changes in the disease pipeline landscape.

Key highlights from the Pompe Disease Pipeline Insight Report
• The Pompe Disease Pipeline report offers a rich analysis of 15+ key players and 20+ key therapies.
• Pompe Disease pipeline comprises therapies in different stages of the clinical phase such as include Cipaglucosidase alfa, SPK-3006, ACTUS 101, AT845, RP A501, AOC-Pompe disease, AVR RD 03, GYS1 Program, ETV-GAA, and many others expected to enter the Pompe Disease market in the coming years.
• Key companies strengthening the Pompe Disease Pipeline are Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics among others.
• The Pompe Disease Companies and academics are working to assess challenges and seek opportunities that could influence Pompe Disease R&D. The therapies under development are focused on novel approaches to treat/improve Pompe Disease.

Recent Developmental Activities in the Pompe Disease Pipeline
• In May 2021, Amicus Therapeutics announced the completion of a successful Type B Pre-Biologics License Application (BLA) meeting with the US Food and Drug Administration (FDA) for AT-GAA (cipaglucosidase alfa co-administered with miglustat), for the treatment of Pompe disease.
• In March 2021, Maze Therapeutics revealed its first three lead therapeutic candidates in the company's wholly owned pipeline. One of the candidates includes an oral therapy targeting GYS1 for the treatment of Pompe disease, enabled by Maze's COMPASS platform. The company expects to start clinical trials in the first half of 2022.

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Pompe Disease Overview
Pompe disease, a severe metabolic myopathy, is caused by mutations in the gene coding for acid alpha-glucosidase (GAA), the enzyme that breaks down glycogen in acidic milieu of the lysosome. Once in the lysosome, glycogen can escape following complete degradation by GAA in the form of glucose. A deficiency of the enzyme leads to lysosomal accumulation of glycogen in multiple tissues, but cardiac and skeletal muscles are most severely affected. The disease also goes by the name "Type II glycogen storage disease (GSDII)" or "Acid maltase deficiency." It is named after a Dutch pathologist, Johannes Cassianus Pompe, who described an autopsy of a 7-month-old girl diagnosed with "idiopathic myocardial hypertrophy" and generalized muscle weakness.

Pompe Disease Emerging Drugs

AT-GAA: Amicus Therapeutics
AT-GAA developed by Amicus Therapeutics is an investigational therapy that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone. In Feb 2019, the US Food and Drug Administration (FDA) granted Amicus a Breakthrough Therapy Designation to AT-GAA for the treatment of late-onset Pompe disease.

SPK-3006: Spark Therapeutics
SPK-3006 developed by Spark Therapeutics is an investigational Pompe disease gene therapy for the potential treatment of the rare condition. Pompe disease is an oftentimes fatal lysosomal storage disorder and neuromuscular disease, with systemic, multi-organ manifestations resulting from loss of function mutations in the gene encoding acid alpha-glucosidase (GAA). The initial construct for SPK-3006 was in-licensed from Genethon in 2017, and Spark retains global commercialization rights.

Pompe Disease Pipeline Therapeutic Analysis
There are approx. 15+ key companies which are developing the therapies for Pompe Disease. The companies which have their Pompe Disease drug candidates in the most advanced stage, i.e. preregistration include Amicus Therapeutics.

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Pompe Disease Pipeline Therapeutic Assessment
The Pompe Disease Pipeline report lays down complete insights into active Pompe Disease pipeline therapies segmented by Stage, Product Type, Route of Administration, Molecule Type, Target, and Mechanism of Action.

Scope of the Pompe Disease Pipeline Report
• Coverage: Global
• Key Players: Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Abeona Therapeutics, AVROBIO, Maze Therapeutic, Denali Therapeutics, Immusoft, Avidity Biosciences, and others.
• Key Pompe Disease Pipeline Therapies: Cipaglucosidase alfa, SPK-3006, ACTUS 101, AT845, RP A501, AIM vectors, AVR RD 03, GYS1 Program, ETV-GAA, Research programme: glycogen storage disease type II therapy, AOC-Pompe disease, and others.

Table of Content
1. Introduction
2. Pompe Disease Executive Summary
3. Pompe Disease: Overview
4. Pompe Disease Pipeline Therapeutics
5. Pompe Disease Therapeutic Assessment
6. Pompe Disease- DelveInsight's Analytical Perspective
7. Late Stage Products (Preregistration)
8. Cipaglucosidase Alfa: Amicus Therapeutics
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. SPK-3006: Spark Therapeutics
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. ACTUS-101: Asklepios Biopharmaceutical
15. Drug profiles in the detailed report…..
16. Early Stage Products (Phase I)
17. Drug name: Company name
18. Drug profiles in the detailed report…..
19. Preclinical and Discovery Stage Products
20. Drug name: Company name
21. Drug profiles in the detailed report…..
22. Inactive Products
23. Pompe Disease Key Companies
24. Pompe Disease Key Products
25. Pompe Disease- Unmet Needs
26. Pompe Disease- Market Drivers and Barriers
27. Pompe Disease- Future Perspectives and Conclusion
28. Pompe Disease Analyst Views
29. Pompe Disease Key Companies
30. Appendix

Key Questions
Current Treatment Scenario and Emerging Therapies:
• How many companies are developing Pompe Disease drugs?
• How many Pompe Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Pompe Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Pompe Disease therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Pompe Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

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This release was published on openPR.