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.
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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.
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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.
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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
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Table of Content
- Introduction
- Executive Summary
- Pompe Disease: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Pompe Disease– DelveInsight’s Analytical Perspective
- Late Stage Products (Preregistration)
- Cipaglucosidase Alfa: Amicus Therapeutics
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- SPK-3006: Spark Therapeutics
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- ACTUS-101: Asklepios Biopharmaceutical
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Drug name: Company name
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug name: Company name
- Drug profiles in the detailed report…..
- Inactive Products
- Pompe Disease Key Companies
- Pompe Disease Key Products
- Pompe Disease- Unmet Needs
- Pompe Disease- Market Drivers and Barriers
- Pompe Disease- Future Perspectives and Conclusion
- Pompe Disease Analyst Views
- Pompe Disease Key Companies
- Appendix
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