Pompe Disease Pipeline Insight

DelveInsight’s, “Pompe Disease – Pipeline Insights, 2022,” 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. 

Geography Covered

  • Global coverage

Pompe Disease Understanding

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 - Pipeline Insight, 2022" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Pompe Disease pipeline landscape is provided which includes the disease overview and Pompe Disease treatment guidelines. The assessment part of the report embraces, in depth Pompe Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Pompe Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

  • The 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. 
  • 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.

Pompe Disease Emerging Drug Chapters

This segment of the Pompe Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

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.

Further product details are provided in the report……..

Pompe Disease: Therapeutic Assessment

This segment of the report provides insights about the Pompe Disease drugs segregated based on following parameters that define the scope of the report, such as:

 

Major Players in Pompe Disease

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.

 

Phases

DelveInsight’s 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) along with the details of 
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates

 

Route of Administration

Pompe Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as 

  • Intranasal
  • Intrathecal
  • Intravenous
  • Oral
  • Oral/Intravenous
  • Parenteral
  • Subcutaneous
  • Subcutaneous/Intramuscular
  • Transdermal

 

Molecule Type

Products have been categorized under various Molecule types such as

  • Antisense oligonucleotide
  • Gene therapy
  • Hormones
  • Neuropeptides
  • Oligonucleotides
  • Small Molecule
  • Triglyceride

 

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Pompe Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Pompe Disease therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Pompe Disease drugs.

Report Highlights

  • The 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.
  • In February 2019, the U.S. Food and Drug Administration (FDA) granted Amicus a Breakthrough Therapy Designation to ATB200/AT2221 for the treatment of late onset Pompe disease.
  • In November 2019 ATB200/AT2221 was granted a PIM by the MHRA. Orphan designations have been granted by the European Commission for both ATB200 and AT2221 in the treatment of glycogen storage disease type II (Pompe disease).
  • In September 2017, ATB200/AT2221was granted US orphan drug status for the treatment of Pompe disease.

Pompe Disease Report Insights

  • Pompe Disease Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Pompe Disease Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

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?

Key Players

  • Immusoft
  • Asklepios Biopharmaceutical
  • Audentes Therapeutics
  • Genzyme
  • Lacerta Therapeutics
  • Selecta Biosciences
  • Greenovation Biotech
  • BioMarin Pharmaceutical
  • Oxyrane

Key Products

  • Cipaglucosidase alfa
  • SPK-3006
  • Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
  • Reveglucosidase alfa
  • Research programme: lysosomal storage disease therapeutics
  • Research programme: alpha-glucosidase gene therapy

 

 

Introduction

Executive Summary

Pompe Disease: Overview

  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics

  • Comparative Analysis

Therapeutic Assessment

  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

Pompe Disease– DelveInsight’s Analytical Perspective

Late Stage Products (Preregistration)

  • Comparative Analysis

Cipaglucosidase Alfa: Amicus Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

SPK-3006: Spark Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I/II)

  • Comparative Analysis

ACTUS-101: Asklepios Biopharmaceutical

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

Drug name: Company name

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical and Discovery Stage Products

  • Comparative Analysis

Drug name: Company name

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

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

List of Table

Table 1: Total Products for Pompe Disease

Table 2: Late Stage Products

Table 3: Mid Stage Products

Table 4: Early Stage Products

Table 5: Pre-clinical & Discovery Stage Products

Table 6: Assessment by Product Type

Table 7: Assessment by Stage and Product Type

Table 8: Assessment by Route of Administration

Table 9: Assessment by Stage and Route of Administration

Table 10: Assessment by Molecule Type

Table 11: Assessment by Stage and Molecule Type                                              

Table 12: Inactive Products

List of Figures

Figure 1: Total Products for Pompe Disease

Figure 2: Late Stage Products                             

Figure 3: Mid Stage Products

Figure 4: Early Stage Products

Figure 5: Preclinical and Discovery Stage Products

Figure 6: Assessment by Product Type                                                                       

Figure 7: Assessment by Stage and Product Type

Figure 8: Assessment by Route of Administration

Figure 9: Assessment by Stage and Route of Administration

Figure 10: Assessment by Molecule Type

Figure 11: Assessment by Stage and Molecule Type

Figure 12: Inactive Products

• Asklepios Biopharmaceutical
• Astellas Pharma Inc
• BioMarin Pharmaceutical
• Amicus Therapeutics
• Spark Therapeutics
• Maze Therapeutics
• Denali Therapeutics
• Rocket Pharmaceuticals
• Audentes Therapeutics

 

 

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