Aplastic Anemia Market

Market Overview:

The 7 major aplastic anemia markets reached a value of USD 301.6 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach USD 567.4 Million by 2035, exhibiting a growth rate (CAGR) of 5.96% during 2025-2035.

The aplastic anemia market has been comprehensively analyzed in IMARC's new report titled "Aplastic Anemia Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2025-2035". Aplastic anemia is a rare, but potentially life-threatening, bone marrow failure disorder that results in a lack of red blood cells, white blood cells, and platelets. The hematologic disorder affects the body's production of adequate blood cells, and its symptoms include severe fatigue, frequent infections, easy bruising, prolonged bleeding, dizziness, and shortness of breath. The condition may be idiopathic or secondary, and its causes include autoimmune diseases, toxic chemical intoxication, radiation, viral diseases, and drugs. Severe forms predispose to life-threatening complications, which need urgent treatment. Diagnosis includes a combination of blood examinations, bone marrow biopsy, and complex genetic and immunological studies. Complete blood count (CBC) demonstrates pancytopenia, and a bone marrow biopsy proves hypocellularity. Additional testing serves to exclude other hematologic disorders and establish the etiology, which is important for directing treatment, such as hematopoietic stem cell transplantation (HSCT) or immunosuppressive therapy.

The increasing rate of aplastic anemia, which is caused by growing exposure to environmental pollutants, viral infections, and autoimmune diseases, is a major driver of market growth. Industrial and agricultural chemicals, such as benzene and pesticides, have been associated with bone marrow depression, causing the increased disease prevalence. Also, viral illnesses like hepatitis, Epstein-Barr virus, and cytomegalovirus are known to cause immune-mediated bone marrow cell destruction leading to additional disease burden. Increased usage of hematopoietic stem cell transplantation (HSCT) as a curative mode of treatment for severe aplastic anemia is driving market growth. Increased transplant success rates due to progress in conditioning regimens and donor-matching methods have made the process more accessible. For patients who are not transplant-eligible, immunosuppressive therapy (IST), such as anti-thymocyte globulin (ATG) and cyclosporine, is still the gold standard, guaranteeing control of the disease. Additionally, the introduction of thrombopoietin (TPO) receptor agonists eltrombopag further increased the production of platelets, minimizing the dependency on transfusions. Ongoing gene therapy and targeted biologics research will further fuel innovation, overcoming current limitations in treatment and broadening the market further.

IMARC Group's new report provides an exhaustive analysis of the aplastic anemia market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, Asia Pacific has the largest patient pool for aplastic anemia and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the aplastic anemia market in any manner.

Recent Developments:

• In December 2024, a Spanish research group proved the safety and effectiveness of gene therapy in the treatment of Fanconi anemia, a genetic type of AA. By repairing the faulty FANCA gene in patients' stem cells and reinfusing them, the altered cells were able to replace the faulty ones in the bone marrow. This method provides a promising alternative to conventional bone marrow transplants, which may lower risks involved and enhance patient quality of life.
• In March 2024, an extension study on romiplostim, a thrombopoietin receptor agonist, assessed its long-term efficacy and safety in patients with refractory AA for a median treatment duration of about 137 weeks. The results presented sustained hematologic responses with tolerable adverse effects, affirming romiplostim as an effective long-term treatment option in refractory AA cases.
• In 2023, equine anti-thymocyte globulin (ATG) was reintroduced for use in Japan, providing additional immunosuppressive therapy options for AA patients. Equine ATG, along with cyclosporine, has been a mainstay in AA treatment, and its availability offers clinicians greater flexibility in treating the disease.

Key Highlights:

• AA is rare, with incidence rates of ~2 per million in Western countries but higher in Asia, reaching 7 per million annually in China.
• The disease peaks at ages 15–25 and over 60, but in India, patients present younger, with a median age of 25 years.
• Western data show an equal male-to-female ratio, but India reports male predominance, possibly due to healthcare access disparities.
• Untreated AA has high mortality; 1-year survival is 82%, dropping to 32% at 3 years, with better outcomes in younger patients.
• Asia reports higher AA incidence (up to 8.8 per million), likely influenced by environmental, genetic, and diagnostic factors.

Drugs:

Novartis' Promacta (Eltrombopag) is an oral thrombopoietin receptor agonist indicated for severe aplastic anemia. It induces platelet production by activating the thrombopoietin pathway, minimizing transfusion dependence. Administered in patients refractory to immunosuppressive therapy, it increases hematopoiesis, enhancing blood cell counts and disease control.

Kyowa Kirin-Amgen's epoetin alfa is a recombinant human erythropoietin, which is developed to induce erythropoiesis in aplastic anemia patients. It increases red cell production, alleviating transfusion dependence. Targeting erythropoietin deficiency, it corrects anemia-related fatigue and enhances the quality of life. Its efficacy and safety in hematologic disorders are well proven.

Hetrombopag, created by Jiangsu Hengrui Medicine Co., is an oral thrombopoietin receptor agonist (TPO-RA) that is meant to treat aplastic anemia. It promotes platelet production through stimulation of the TPO receptor to counter thrombocytopenia in bone marrow failure patients. Hetrombopag presents a promising treatment choice for enhancing blood cell counts in aplastic anemia.

REGN7257 is a therapeutic monoclonal antibody that is aimed against the interleukin-2 receptor subunit gamma (IL2RG). It is being evaluated in Phase 1/2 clinical trials to evaluate its safety and efficacy for the treatment of patients with severe aplastic anemia (SAA) who are refractory or have relapsed after immunosuppressive therapy.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2025-2035

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the aplastic anemia market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the aplastic anemia market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current aplastic anemia marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the aplastic anemia market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2035?
• What was the country-wise size of the aplastic anemia market across the seven major markets in 2024 and what will it look like in 2035?
• What is the growth rate of the aplastic anemia market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?

Epidemiology Insights

• What is the number of prevalent cases (​2019-2035) of aplastic anemia across the seven major markets?
• What is the number of prevalent cases (​2019-2035​) of aplastic anemia by age across the seven major markets?
• What is the number of prevalent cases (​2019-2035) of aplastic anemia by gender across the seven major markets?
• How many patients are diagnosed (​2019-2035) with aplastic anemia across the seven major markets?
• What is the size of the aplastic anemia patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2025-2035) across the seven major markets?
• What are the key factors driving the epidemiological trend of aplastic anemia?
• What will be the growth rate of patients across the seven major markets?

Aplastic Anemia: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for aplastic anemia drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the aplastic anemia market?
• What are the key regulatory events related to the aplastic anemia market?
• What is the structure of clinical trial landscape by status related to the aplastic anemia market?
• What is the structure of clinical trial landscape by phase related to the aplastic anemia market?
• What is the structure of clinical trial landscape by route of administration related to the aplastic anemia market?