Hemophilia A Pipeline Insight

DelveInsight’s, “Hemophilia A - Pipeline Insight, 2021,” report provides comprehensive insights about 45+ companies and 45+ pipeline drugs in Hemophilia A 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

Hemophilia A Understanding

Hemophilia A: Overview

Hemophilia A, also called factor VIII (8) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII (FVIII), a clotting protein. Hemophilia is inherited in an X-linked recessive manner. Hemophilia can vary in its severity, depending upon the particular type of mutation (genetic defect). The degree of symptoms depends upon the levels of the affected clotting factor. Symptoms of Hemophilia A include excessive bleeding from any site in the body along with long-term damage to joints from repeated bleeding episodes is characteristic. Diagnosis for suspected hemophilia include tests like Complete blood cell count, Screening coagulation studies, FVIII assay and FVIII inhibitor assay. Treatment goal for hemophilia is to replace the missing blood clotting factor so that the blood can clot properly. Medication to treat hemophilia A is concentrated FVIII product. Several new treatments for hemophilia A are under development, from gene therapy to new non-factor replacement therapies.


"Hemophilia A - Pipeline Insight, 2021" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hemophilia A pipeline landscape is provided which includes the disease overview and Hemophilia A treatment guidelines. The assessment part of the report embraces, in depth Hemophilia A 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, Hemophilia A 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 Hemophilia A R&D. The therapies under development are focused on novel approaches to treat/improve Hemophilia A.

Hemophilia A Emerging Drugs Chapters

This segment of the Hemophilia A report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.


Hemophilia A Emerging Drugs


Fitusiran: Alnylam Pharmaceuticals/Sanofi Genzyme

Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT). Alnylam Pharmaceuticals is developing the drug for the treatment of hemophilia and rare bleeding disorders (RBDs) in collaboration with Sanofi Genzyme. Fitusiran utilizes Alnylam Pharmaceuticals’ Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform. Fitusiran has been granted Orphan Drug Designation by FDA and Orphan Medicinal Product Designation by EMA for the treatment of hemophilia A and B.


Concizumab: Novo Nordisk

Concizumab is a high-affinity, humanized, anti–tissue factor (TF) pathway inhibitor (TFPI) based monoclonal antibody. The therapy is in clinical evaluation as a subcutaneously administered treatment for hemophilia A and B.

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

Hemophilia A: Therapeutic Assessment

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

  • Major Players in Hemophilia A

There are approx. 45+ key companies which are developing the therapies for Hemophilia A. The companies which have their Hemophilia A drug candidates in the most advanced stage, i.e. Phase III include, Novo Nordisk.

Phases

DelveInsight’s report covers around 45+ 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


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

  • Oral
  • Parenteral
  • Intravitreal
  • Subretinal
  • Topical
  • Molecule Type


Products have been categorized under various Molecule types such as

  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type

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

Hemophilia A: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Hemophilia A 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 Hemophilia A drugs.

Hemophilia A Report Insights

  • Hemophilia A Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Hemophilia A 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 Hemophilia A drugs?
  • How many Hemophilia A drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hemophilia A?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hemophilia A 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 Hemophilia A and their status?
  • What are the key designations that have been granted to the emerging drugs?

Introduction

Executive Summary

Hemophilia A: 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 

Hemophilia A – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

  • Hemophilia A companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Hemophilia A Collaboration Deals

  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products (Phase III)

  • Comparative Analysis

Fitusiran: Alnylam Pharmaceuticals/Sanofi Genzyme

  • Product Description
  • Research and Development 
  • Product Development Activities 

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

Drug name: Company name

  • Product Description
  • Research and Development 
  • Product Development Activities 

Drug profiles in the detailed report…..

Early Stage Products (Phase I/II) 

  • Comparative Analysis

DTX 201: Dimension Therapeutics

  • Product Description
  • Research and Development 
  • Product Development Activities 

Drug profiles in the detailed report…..

Preclinical and Discovery Stage Products

  • Comparative Analysis

KY1049: Kymab

  • Product Description
  • Research and Development 
  • Product Development Activities 

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Hemophilia A Key Companies

Hemophilia A Key Products

Hemophilia A- Unmet Needs

Hemophilia A- Market Drivers and Barriers

Hemophilia A- Future Perspectives and Conclusion

Hemophilia A Analyst Views

Hemophilia A Key Companies

Appendix

List of Table

Table 1: Total Products for Hemophilia A

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 Hemophilia A

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      


• Alnylam Pharmaceuticals

• Sanofi Genzyme

• Novo Nordisk

• Catalyst Biosciences

• Dimension Therapeutics

• Chia Tai Tianqing Pharmaceutical Group

• Staidson Beijing BioPharmaceuticals

• Kymab

• Poseida Therapeutics

• Intellia Therapeutics

• Regeneron Pharmaceuticals

• Hoffmann-La Roche

• Spark Therapeutics

• BioMarin

• Centessa Pharmaceuticals

• Expression Therapeutics

• Chugai Pharmaceutical

• Pfizer

  • Tags:
  • Hemophilia A Pipeline
  • Hemophilia A clinical trials
  • Hemophilia A companies
  • Hemophilia A Drugs
  • Hemophilia A Therapies
  • Hemophilia A Treatment algorithm
  • Hemophilia A Pipeline drugs
  • Hemophilia A Pipeline companies

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