DelveInsight’s “Aplastic Anemia Treatment Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of Aplastic Anemia, historical and forecasted epidemiology, as well as the Aplastic Anemia market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

Discover which therapies are expected to grab the Aplastic Anemia Market Share @ Aplastic Anemia Market Outlook

Key Takeaways from the Aplastic Anemia Market Report

• In May 2025, Emory University announced a study will enroll 30 pediatric patients with serious non-malignant hematologic diseases (NMHD) undergoing URD HSCT. The trial will include patients with 7/8 donors and those with 8/8 (matched) donors. All participants will receive 8 doses of abatacept. Recruitment is expected to last for about 2 years and participants will be followed for up to 3 years.
• In May 2025, Medical College of Wisconsin conducted a study is a prospective, multicenter Phase II study of hematopoietic stem cell transplantation for previously untreated patients with severe aplastic anemia (SAA). Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. Patients with aplastic anemia have low white blood cells (cells which fight infection), low red blood cells (cells that carry oxygen throughout the body), and low platelets (cells that help form clots and prevent bleeding). Treatments for SAA seeks to repair this abnormal immune system attack and allow the bone marrow to make the normal amount of blood cells. This can be done with a bone marrow transplant or with medications to suppress the immune system.
• In May 2025, Fred Hutchinson Cancer Center organized 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. Acquired aplastic anemia (AA) is a condition in which the bone marrow is unable to produce blood cells.
• 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 PF-06462700, REGN7257, BL-8040, and Others.

Stay ahead in the Aplastic Anemia Therapeutics Market with DelveInsight’s Strategic Report @ Aplastic Anemia Treatment Market

Aplastic Anemia Epidemiology Segmentation in the 7MM

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

Download the report to understand which factors are driving Aplastic Anemia Epidemiology trends @ Aplastic Anemia Prevalence

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.

To learn more about Aplastic Anemia Treatment guidelines, visit @ Aplastic Anemia Clinical Trials Assessment

Aplastic Anemia Companies

Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals and others.

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

Because newly authorized Aplastic Anemia drugs are often expensive, some patients escape receiving proper treatment or use off-label, less expensive prescriptions. Reimbursement plays a critical role in how innovative treatments can enter the market. The cost of the medicine, compared to the benefit it provides to patients who are being treated, sometimes determines whether or not it will be reimbursed. Regulatory status, target population size, the setting of treatment, unmet needs, the number of incremental benefit claims, and prices can all affect market access and reimbursement possibilities.

Learn more about the FDA-approved drugs for Aplastic Anemia @ Drugs for Aplastic Anemia Treatment

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- PF-06462700, REGN7257, BL-8040, and Others.
• Aplastic Anemia Market Dynamics: Aplastic Anemia Market Drivers and Barriers
• Aplastic Anemia Market Access and Reimbursement, Unmet Needs and Future Perspectives

Table of Contents

1. Key Insights

2. Report Introduction

3. Executive Summary of Aplastic Anemia

4. Aplastic Anemia Market Overview at a Glance

5 Key Events

6 Epidemiology and Market Forecast Methodology

7 Disease Background and Overview

8 Treatment and Management of Aplastic Anemia

9 Epidemiology and Patient Population

10 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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