Aplastic anemia Market: Epidemiology, Therapies, Companies, DelveInsight | Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals

Aplastic anemia therapies, such as PF-06462700, REGN7257, BL-8040, and others, are expected to boost the Aplastic anemia Market in the upcoming years.

DelveInsight has launched a new report on "Aplastic anemia - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Aplastic anemia, historical and forecasted epidemiology as well as the Aplastic anemia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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Some of the key facts of the Aplastic anemia Market Report:

In 2023, the United States represented the largest share of the Aplastic Anemia treatment market, accounting for nearly 70% of the total market across the seven major markets (7MM), surpassing the EU4, the UK, and Japan. Aplastic Anemia is a rare and serious non-cancerous condition marked by the immune system attacking early blood-forming cells. Its global incidence ranges from 0.7 to 7.4 cases per million people annually, with significantly higher rates observed in Asia compared to Europe and the U.S.
Severe and very severe cases make up roughly 65% to 80% of the total Aplastic Anemia patient population. Treatment choice depends heavily on age and disease severity-hematopoietic stem cell transplantation (HSCT) is the preferred option for patients under 40, while immunosuppressive therapy (IST) remains the standard for older patients. Younger individuals tend to have favorable long-term outcomes, whereas prognosis in patients aged 60 and above remains poor, indicating a critical need for better treatment options in older populations.
Among pharmacologic treatments, Novartis' PROMACTA (known as REVOLADE in some regions) leads in revenue generation, followed by immunosuppressive therapies. PROMACTA's sales are primarily driven by its use in chronic immune thrombocytopenic purpura (ITP) and severe aplastic anemia (SAA). Despite the introduction of new therapies and recent approvals, the market is still largely propelled by ISTs and PROMACTA/REVOLADE.
Several key companies are pursuing innovative therapies: Regeneron Pharmaceuticals is advancing REGN7257 for patients with refractory or relapsed SAA following IST, while Gamida Cell is developing cord blood-derived stem and progenitor cell therapies to improve transplant outcomes. Research is also exploring the potential of combining androgens and growth factors such as G-CSF and erythropoietin with standard regimens to enhance bone marrow recovery and blood cell production.
Despite these advancements, significant challenges remain-particularly in developing effective, well-tolerated treatments for patients unresponsive to existing therapies or those unsuitable for intensive procedures. Notably, among the 7MM, Japan recorded the highest number of incident Aplastic Anemia cases in 2023, accounting for around 30% of the total.
In April 2025, the FDA granted Orphan Drug Designation to CK0801, an allogeneic cord blood-derived T regulatory (Treg) cell therapy developed by Cellenkos Inc., for the treatment of aplastic anemia. This designation aims to expedite the development of therapies for rare diseases. CK0801 is currently planned for a registration trial in the second half of 2025.
Key Aplastic anemia companies such as Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, and others are evaluating new drugs for Aplastic anemia to improve the treatment landscape.
Promising Aplastic anemia therapies include PF-06462700, REGN7257, BL-8040, and others.

Aplastic anemia Overview

Myositis refers to inflammation of the muscles and is commonly linked with a group of inflammatory muscle diseases, including polymyositis, dermatomyositis, cancer-associated myositis, juvenile dermatomyositis, overlap myositis, and inclusion body myositis (IBM). While *myositis* specifically involves inflammation, *myopathy* is a broader term encompassing any disease or disorder of the muscles, including but not limited to myositis.

This rare condition can impact not only the muscles but also various other organs, often resulting in significant reductions in quality of life. According to the American College of Rheumatology, inflammatory myopathies are autoimmune diseases in which the immune system mistakenly attacks healthy muscle tissue. Dermatomyositis and polymyositis are the most frequently occurring types within this group.

Aplastic anemia Market Outlook

The prognosis for aplastic anemia varies depending on the disease's severity. While some patients respond well to treatment, others may develop serious complications such as myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML).

Traditionally, the main treatment approaches have included immunosuppressive therapy (IST) and bone marrow transplantation (BMT) for eligible patients. For those with severe or very severe aplastic anemia (SAA/VSAA) who are candidates for transplant, age plays a crucial role in determining survival outcomes following matched sibling donor (MSD) allogeneic transplantation. Older patients, who often have prior IST exposure, coexisting medical conditions, poorer performance status, and delays in receiving BMT, tend to face more complex treatment challenges. In this group, fludarabine-based regimens have shown some promise, although the supporting evidence is mostly limited to retrospective studies.

In clinical practice, the management of non-severe aplastic anemia (NSAA) differs notably from that of SAA/VSAA. HSCT is not used for NSAA, and treatment initiation criteria are less well established. Additionally, there is a lack of standardized guidelines for assessing and treating NSAA, with care typically depending on the experience and protocols of individual treatment centers.

As several novel therapies are currently under investigation, the aplastic anemia treatment landscape is expected to undergo significant transformation between 2024 and 2034.

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Aplastic Anemia Marketed Therapies
ALVAIZ (eltrombopag choline): Teva Pharmaceuticals
ROMIPLATE (romiplostim; AMG531): Kyowa Kirin/Amgen
Aplastic anemia Emerging Drugs
REGN7257: Regeneron Pharmaceuticals
OMISIRGE (omidubicel): Gamida Cell

Scope of the Aplastic anemia Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Aplastic anemia Companies: Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, and others
Key Aplastic anemia Therapies: PF-06462700, REGN7257, BL-8040, and others
Aplastic anemia Therapeutic Assessment: Aplastic anemia current marketed and Aplastic anemia emerging therapies
Aplastic anemia Market Dynamics: Aplastic anemia market drivers and Aplastic anemia market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
Aplastic anemia Unmet Needs, KOL's views, Analyst's views, Aplastic anemia Market Access and Reimbursement

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Key benefits of the Aplastic anemia Market Report:
Aplastic anemia market report covers a descriptive overview and comprehensive insight of the Aplastic anemia Epidemiology and Aplastic anemia market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
The Aplastic anemia market report provides insights into the current and emerging therapies.
The Aplastic anemia market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Aplastic anemia market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Aplastic anemia market.

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Table of Contents

1. Report Introduction
2. Executive Summary
3. SWOT analysis
4. Aplastic anemia Patient Share (%) Overview at a Glance
5. Aplastic anemia Market Overview at a Glance
6. Aplastic anemia Disease Background and Overview
7. Aplastic anemia Epidemiology and Patient Population
8. Country-Specific Patient Population of Aplastic anemia
9. Aplastic anemia Current Treatment and Medical Practices
10. Unmet Needs
11. Aplastic anemia Emerging Therapies
12. Aplastic anemia Market Outlook
13. Country-Wise Aplastic anemia Market Analysis (2020-2034)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Aplastic anemia Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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Aplastic anemia Pipeline Insights, DelveInsight

"Aplastic anemia Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Aplastic anemia market. A detailed picture of the Aplastic anemia pipeline landscape is provided, which includes the disease overview and Aplastic anemia treatment guidelines.

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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.

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