The Alpha Thalassemia Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

“Alpha Thalassemia Pipeline Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Alpha Thalassemia Market.

Some of the key takeaways from the Alpha Thalassemia Pipeline Report:

• Companies across the globe are diligently working toward developing novel Alpha Thalassemia treatment therapies with a considerable amount of success over the years.

• Alpha Thalassemia companies working in the treatment market are Forma Therapeutics, Inc., Genmedicn Biopharma Ltd., Bristol-Myers Squibb, Agios Pharmaceuticals, and others, are developing therapies for the Alpha Thalassemia treatment

• Emerging Alpha Thalassemia therapies in the different phases of clinical trials are- Etavopivat tablets, GMCN-508A, Luspatercept, Mitapivat, and others are expected to have a significant impact on the Alpha Thalassemia market in the coming years.

• In January 2025, Agios Pharmaceuticals (AGIO) revealed that the FDA has accepted its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) as a treatment for adult patients with both transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. The FDA has granted the application a standard review designation, setting a target decision date of September 7, 2025, under the Prescription Drug User Fee Act (PDUFA).

• In December 2024, Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation, which is pioneering therapies for rare diseases, today announced positive results from the Phase 3 ENERGIZE-T study of mitapivat, an oral small-molecule PK activator, in adults with transfusion-dependent alpha- or beta-thalassemia. These results were presented in an oral session (abstract #409) at the 66th Annual Meeting and Exposition of the American Society of Hematology (ASH) in San Diego, California.

• In July 2024, Researchers from Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine have developed an innovative model that could serve as a platform for creating new therapies to treat Alpha Thalassemia (AT), a serious blood disorder. Their findings were published in the journal Blood. Each year, thousands of children are born with AT, particularly in regions like Southeast Asia, India, the Middle East, and the Mediterranean. While the genetic trait can offer protection against malaria and cause only mild anemia in its normal form, children whose parents both carry defective genes face a significantly higher risk of severe AT.

• In January 2024, Agios Pharmaceuticals reported positive outcomes in a Phase III ENERGIZE trial of mitapivat for patients with non-transfusion-dependent alpha- or beta-thalassemia, meeting primary and two key secondary endpoints. The company intends to seek regulatory approval for mitapivat as a thalassemia treatment by year-end, incorporating data from the second Phase III ENERGISE-T study (NCT04770779) for adults with transfusion-dependent alpha-thalassemia or beta-thalassemia.

Alpha Thalassemia Overview

Alpha thalassemia is a genetic disorder characterized by a deficiency in the production of alpha globin chains, which are essential components of hemoglobin, the protein in red blood cells that carries oxygen throughout the body. This deficiency results in a reduced amount of hemoglobin and red blood cells, leading to anemia.

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Emerging Alpha Thalassemia Drugs Under Different Phases of Clinical Development Include:

• Etavopivat tablets: Forma Therapeutics, Inc.

• GMCN-508A: Genmedicn Biopharma Ltd.

• Luspatercept: Bristol-Myers Squibb

• Mitapivat: Agios Pharmaceuticals

Alpha Thalassemia Route of Administration

Alpha Thalassemia 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

• Intravenous

• Subcutaneous

• Topical

Alpha Thalassemia Molecule Type

Alpha Thalassemia Products have been categorized under various Molecule types, such as

• Monoclonal Antibody

• Peptides

• Polymer

• Small molecule

• Gene therapy

• Product Type

Alpha Thalassemia Pipeline Therapeutics Assessment

• Alpha Thalassemia Assessment by Product Type

• Alpha Thalassemia By Stage and Product Type

• Alpha Thalassemia Assessment by Route of Administration

• Alpha Thalassemia By Stage and Route of Administration

• Alpha Thalassemia Assessment by Molecule Type

• Alpha Thalassemia by Stage and Molecule Type

DelveInsight’s Alpha Thalassemia Report covers around 5+ products under different phases of clinical development like

• Late-stage products (Phase III)

• Mid-stage products (Phase II)

• Early-stage product (Phase I)

• Pre-clinical and Discovery stage candidates

• Discontinued & Inactive candidates

• Route of Administration

Further Alpha Thalassemia product details are provided in the report. Download the Alpha Thalassemia pipeline report to learn more about the emerging Alpha Thalassemia therapies

Alpha Thalassemia Pipeline Analysis:

The Alpha Thalassemia pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Alpha Thalassemia with aggregate therapies developed by each company for the same.

• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Alpha Thalassemia Treatment.

• Alpha Thalassemia key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• Alpha Thalassemia Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.

• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Alpha Thalassemia market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Alpha Thalassemia drugs and therapies

Alpha Thalassemia Pipeline Market Drivers

• Increasing Prevalence genetic disorders and advancement in healthcare technologies, growing demand for treatment for Alpha Thalassemia are some of the important factors that are fueling the Alpha Thalassemia Market.

Alpha Thalassemia Pipeline Market Barriers

• However, lack of available appropriate treatments and procedures, high cost of treatment and other factors are creating obstacles in the Alpha Thalassemia Market growth.

Scope of Alpha Thalassemia Pipeline Drug Insight

• Coverage: Global

• Key Alpha Thalassemia Companies: Forma Therapeutics, Inc., Genmedicn Biopharma Ltd., Bristol-Myers Squibb, Agios Pharmaceuticals, and others

• Key Alpha Thalassemia Therapies: Etavopivat tablets, GMCN-508A, Luspatercept, Mitapivat, and others

• Alpha Thalassemia Therapeutic Assessment: Alpha Thalassemia current marketed and Alpha Thalassemia emerging therapies

• Alpha Thalassemia Market Dynamics: Alpha Thalassemia market drivers and Alpha Thalassemia market barriers

Request for Sample PDF Report for Alpha Thalassemia Pipeline Assessment and clinical trials

Table of Contents

1. Alpha Thalassemia Report Introduction

2. Alpha Thalassemia Executive Summary

3. Alpha Thalassemia Overview

4. Alpha Thalassemia- Analytical Perspective In-depth Commercial Assessment

5. Alpha Thalassemia Pipeline Therapeutics

6. Alpha Thalassemia Late Stage Products (Phase II/III)

7. Alpha Thalassemia Mid Stage Products (Phase II)

8. Alpha Thalassemia Early Stage Products (Phase I)

9. Alpha Thalassemia Preclinical Stage Products

10. Alpha Thalassemia Therapeutics Assessment

11. Alpha Thalassemia Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Alpha Thalassemia Key Companies

14. Alpha Thalassemia Key Products

15. Alpha Thalassemia Unmet Needs

16 . Alpha Thalassemia Market Drivers and Barriers

17. Alpha Thalassemia Future Perspectives and Conclusion

18. Alpha Thalassemia Analyst Views

19. Appendix

20. About DelveInsight

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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