DelveInsight’s, “Pompe Disease Pipeline Insight 2024” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in 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.

 

Discover the latest drugs and treatment options in the Pompe Disease Pipeline. Dive into DelveInsight’s comprehensive report today! @ Pompe Disease Pipeline Outlook

 

Key Takeaways from the Pompe Disease Pipeline Report

• July 2024:- Amicus Therapeutics:- This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years.
• July 2024:- Sanofi– An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD).
• DelveInsight’s Pompe Disease pipeline report depicts a robust space with 15+ active players working to develop 20+ pipeline therapies for Pompe Disease treatment.
• The leading Pompe Disease Companies such as Immusoft, Asklepios, Oxyrane, Maze Therapeutics, Amicus Therapeutics, Spark Therapeutics, and others.
• Promising Pompe Disease Therapies such as ATB200, AT2221, GC301, avalglucosidase alfa, SPK-3006, and others.

 

Stay ahead with the most recent pipeline outlook for Pompe Disease. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Pompe Disease Treatment Drugs

 

Pompe Disease Emerging Drugs Profile

 

• 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.

 

Explore groundbreaking therapies and clinical trials in the Pompe Disease Pipeline. Access DelveInsight’s detailed report now! @ New Pompe Disease Drugs

 

Pompe Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

 

Pompe Disease  Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide

 

Unveil the future of Pompe Disease Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight’s expert analysis @ Pompe Disease Market Drivers and Barriers

 

Scope of the Pompe Disease Pipeline Report

• Coverage- Global
• Pompe Disease Companies- Immusoft, Asklepios, Oxyrane, Maze Therapeutics, Amicus Therapeutics, Spark Therapeutics, and others.
• Pompe Disease Therapies- ATB200, AT2221, GC301, avalglucosidase alfa, SPK-3006, and others.
• Pompe Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Pompe Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

 

Get the latest on Pompe Disease Therapies and clinical trials. Download DelveInsight’s in-depth pipeline report today! @ Pompe Disease Companies, Key Products and Unmet Needs

 

Table of Content

1. Introduction
2. Executive Summary
3. Pompe Disease: Overview
4. Pipeline Therapeutics
5. 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

 

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