DelveInsight’s ” Aplastic Anemia Market Insights, Epidemiology, and Market Forecast- 2034” report 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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Key Takeaways from the Aplastic Anemia Market Report

• In April 2025, Fred Hutchinson Cancer Center announced a phase II trial tests how well a ruxolitinib-based graft versus host disease (GVHD) prevention (prophylaxis) regimen works before, during, and after bone marrow/stem cell transplantation (hematopoietic cell transplantation [HCT]) in patients with acquired aplastic anemia.
• In March 2025, Novartis Pharmaceuticals announced a phase II study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric patients with previously untreated or relapsed/refractory severe aplastic anemia or recurrent aplastic anemia.
• The total Aplastic Anemia Treatment Market Size was found to be ~USD 270 million in 2023 in the 7MM, which is further expected to increase by 2034.
• Among the 7MM, Japan accounts for the second highest total aplastic anemia drugs market. The Aplastic Anemia Treatment Market Size in Japan has been assessed to be ~USD 35 million in 2023, expected to increase during the forcast period.
• During the forecast period, Aplastic Anemia pipeline candidates such as Omidubicel-onlv, REGN7257, and ALVAIZ (eltrombopag choline) are expected to drive the rise in Aplastic anemia market size.
• Among EU4 and the UK, Germany accounts for the highest Aplastic Anemia Treatment Market Size with ~USD 14 million in 2023, followed by France.
• Aplastic Anemia Treatment Market Size is expected to increase due to several factors such as an increase in disease incidence in Asian countries, high patient uptake of PROMACTA (until expected patent expiry in 2025), approval of Teva Pharma’s ALVAIZ in the US, and Kyowa Kirin’s ROMIPLATE in Japan along with the expected launch of potential emerging therapies.
• The leading Aplastic Anemia Companies such as Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, and others.
• Promising Aplastic Anemia Pipeline Therapies such as Romiplostim, Eltrombopag, PF-06462700, AMG531, Rabbit ATG, Cyclosporine and others.

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Aplastic Anemia Epidemiology Segmentation in the 7MM

• Aplastic Anemia Incident Cases
• Aplastic Anemia Severity-Specific Cases
• Aplastic Anemia Age-specific Cases

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Aplastic Anemia Marketed Therapies

• ALVAIZ (eltrombopag choline): Teva Pharmaceuticals

ALVAIZ (eltrombopag choline) is an oral thrombopoietin (TPO) receptor agonist that works to increase platelet production by interacting with the transmembrane domain of the human TPO-receptor to initiate signaling cascades that induce proliferation and differentiation of megakaryocytes. Lowest dose of ALVAIZ is use to achieve and maintain a hematologic response. Dose adjustments are based upon the platelet count. Hematologic response requires dose titration, generally up to 108 mg, and may take up to 16 weeks after starting ALVAIZ.

• ROMIPLATE (romiplostim; AMG531): Kyowa Kirin/Amgen

ROMIPLATE is composed of recombinant protein acting on the thrombopoietin receptor, which has been licensed from Amgen (K-A) to Kyowa Kirin. It was launched as a drug for idiopathic thrombocytopenic purpura (ITP) in April 2011 and for aplastic anemia in patients who had an inadequate response to conventional therapy in June 2019 in Japan. The drug is composed of recombinant protein stimulating hematopoiesis via acting on the thrombopoietin receptors; Acts directly on megakaryocytic progenitor cells and exerts platelet hematopoietic effects. In addition, this drug promotes the proliferation and differentiation of megakaryocytic progenitor cells by binding to and activating thrombopoietin receptors.

Aplastic Anemia Emerging Therapies

• REGN7257: Regeneron Pharmaceuticals

It’s a gc cytokine receptor antibody that targets the common g chain (gc; IL-2RG) found in interleukin (IL) receptors for various gc cytokines, including IL-2, IL-4, IL-7, IL-9, IL-15, and IL-21. It was developed to investigate the potential of targeting gc cytokines for the prevention and treatment of T-cell-mediated diseases. By blocking the signaling of GC cytokines, REGN7257 aims to ameliorate T-cell-mediated pathogenesis. This antibody offers a promising approach to understand better the role of gc cytokines in inflammatory and autoimmune disease settings, where the lack of appropriate neutralizing antibodies recognizing IL-2RG has been a challenge.

• OMISIRGE (omidubicel): Gamida Cell

OMISIRGE is a nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood. It is indicated for use in adults and pediatric patients aged 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning. The therapy aims to reduce the time to neutrophil recovery and the incidence of infection in this patient population. Omisirge has received approval for use in hematological malignancies and is currently undergoing evaluation in an ongoing investigator-sponsored Phase I/II study focusing on patients with severe aplastic anemia.

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Aplastic Anemia Market Outlook

The Aplastic Anemia Prognosis, varies based on the severity of the condition. Some cases can be successfully treated, while others may progress to severe complications, such as myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). Historically, the primary modalities for treating aplastic anemia have been IST and BMT in eligible patients. For individuals with SAA/VSAA deemed suitable for transplant-based interventions, age emerges as a pivotal determinant influencing survival post-matched sibling donor (MSD) allogeneic transplantation. Notably, older patients, often having undergone prior IST, presenting with additional comorbidities, diminished performance status, and an extended duration between diagnosis and BMT, exhibit distinctive challenges in outcomes, with fludarabine-containing regimens offering more promising results in this demographic, albeit based on limited retrospective analyses.

Aplastic Anemia Treatment Market

Aplastic anemia is a rare and severe nonmalignant disease characterized by autoimmune destruction of early hematopoietic cells. Aplastic anemia can be categorized into congenital and acquired forms. Inherited forms are rare and include Fanconi Anemia, Congenital Keratosis, Congenital Pure Red Cell Aplasia, and Shwachman–Diamond Syndrome. Most cases of acquired aplastic anemia occur unrelated to any identifiable causes or for unknown reasons. Researchers believe that most are due to the immune system mistakenly targeting the bone marrow. Specific symptoms vary from case to case. Some individuals may have mild symptoms that remain stable for many years; others may have serious symptoms that can progress to life-threatening complications.

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Scope of the Aplastic Anemia Market Report

• Coverage- 7MM
• Study Period- 2020-2034
• Aplastic Anemia Companies- Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, and others.
• Aplastic Anemia Pipeline Therapies- Romiplostim, Eltrombopag, PF-06462700, AMG531, Rabbit ATG, Cyclosporine, and others.
• Aplastic Anemia Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Aplastic Anemia Unmet Needs, KOL’s views, Analyst’s views, Aplastic Anemia Market Access and Reimbursement

Table of Contents

1. Key Insights

2. Aplastic Anemia Market Report Introduction

3. Executive Summary of Aplastic Anemia

4. Aplastic Anemia Market Overview at a Glance

5 Key Events

6 Aplastic Anemia Epidemiology and Market Forecast Methodology

7 Aplastic Anemia Disease Background and Overview

8 Aplastic Anemia Treatment and Management of Aplastic Anemia

9 Aplastic Anemia Epidemiology and Patient Population

10 Aplastic Anemia Patient Journey

11 Aplastic Anemia Marketed Therapies

12 Aplastic Anemia Emerging Drugs

13 Aplastic Anemia: Market Analysis

14 Aplastic Anemia Unmet Needs

15 Aplastic Anemia SWOT Analysis

16 Aplastic Anemia KOL Views

17 Aplastic Anemia Market Access and Reimbursement

18 Appendix

19 DelveInsight Capabilities

20 Disclaimer

21 About DelveInsight

About Us

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