Aplastic Anemia - Market Insight, Epidemiology and Market Forecast - 2034

Key Highlights

• In the 7MM, United States accounted for the largest market size in 2023, with approximately USD ~190 million.
• Aplastic anemia is a rare and severe nonmalignant disease characterized by autoimmune destruction of early hematopoietic cells. Its global incidence rate ranges from 0.7-7.4 cases per million inhabitants per year, with higher rates in Asia compared to Europe and the United States.
• The two main forms of specific treatment are bone marrow transplantation and immunosuppressive therapies. The two most commonly used immunosuppressive agents, given alone or in combination, are antithymocyte globulin (ATG) and cyclosporine. In the context of patients aged 40 years and above, frontline IST stands as the prevailing standard of care. Conversely, BMT remains the preferred therapeutic avenue for children and young adults diagnosed with SAA, particularly those possessing an MSD.
• THYMOGLOBULINE (rabbit ATG) was developed by Sanofi and approved in Japan in 2008 for moderate to VSAA.
• The landscape of therapies for SAA witnessed a transformative moment in 2014 with the approval of PROMACTA (eltrombopag), a thrombopoietin receptor agonist, by the FDA for SAA cases inadequately responsive to initial IST, and later got approved in Europe and Japan in 2015 and 2017, respectively, under the brand name REVOLADE. Subsequently, in 2018, the US FDA approved PROMACTA as a first-line treatment for SAA.
• Another Tpo receptor agonist, ROMIPLATE, was granted approval in Japan in June 2019 for aplastic anemia cases where conventional treatments have proven insufficient. Subsequently, in September 2023, Kyowa Kirin disclosed the expanded approval of ROMIPLATE as a first-line treatment for aplastic anemia in Japan.
• Key players, such as Regeneron Pharmaceuticals, are working on REGN7257 for refractory or relapsed SAA after immunosuppressive therapy, and Gamida Cell is developing umbilical cord blood-derived stem and progenitor cells to enhance engraftment and improve transplant outcomes in unrelated umbilical cord blood transplantation for SAA.
• PROMACTA held the leading market share in the 7MM in 2023, boasting a market size of around USD ~160 million. This figure is projected to experience further growth by the year 2034.
• Despite the advancements in treating aplastic anemia, there remain challenges in finding effective and well-tolerated treatments, especially for patients who do not respond to standard therapies or for those who may not be suitable candidates for more aggressive interventions.
• The unmet need in aplastic anemia continues to be addressed through novel therapies like eltrombopag and ROMIPLATE, but the limited emerging pipeline underscores the necessity for further research and development to expand treatment options for this challenging condition.

This 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, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The aplastic anemia market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM aplastic anemia market size from 2020 to 2034. The report also covers current aplastic anemia treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s potential.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2020-2034

Aplastic Anemia Disease Understanding and Treatment Algorithm

Aplastic Anemia Overview

Aplastic anemia refers to the syndrome of chronic primary hematopoietic failure from injury leading to diminished or absent hematopoietic precursors in the bone marrow and attendant pancytopenia. The symptoms of aplastic anemia occur because the bone marrow fails to produce enough blood cells. Some individuals may have mild symptoms that remain stable for many years; others may have serious symptoms that can progress to life-threatening complications. Individuals with anemia may experience tiredness, increased need for sleep, weakness, lightheadedness, dizziness, irritability, headaches, pale skin color, difficulty breathing, and cardiac symptoms like chest pain.

Aplastic Anemia Diagnosis

The rarity of aplastic anemia, coupled with its overlap with other bone marrow failure syndromes, poses a diagnostic challenge.Except for findings related to bleeding or infections, the examination presents mainly negative characteristics: absence of lymphadenopathy, no enlarged spleen or liver, and no infiltration of any other organ. Bone marrow (BM) aspiration and biopsy are essential procedures for diagnosing aplastic anemia. Cytogenetic abnormalities can be detected in up to 12-15% of otherwise typical aplastic anemia patients, making systematic cytogenetic investigations essential for all aplastic anemia patients. Molecular analysis, including next-generation sequencing (NGS), has become increasingly instrumental in comprehending the pathophysiology of diseases.

Aplastic Anemia Treatment

Treatment of aplastic anemia varies, depending upon the individual’s age, general health, and the severity of aplastic anemia. Treatment aims to correct the bone marrow failure, as well as to treat the patient’s immediate signs and symptoms. The two main forms of specific treatment are bone marrow transplantation and immunosuppressive therapies. Initial treatment of acquired aplastic anemia may be directed toward improving the symptoms that may result from low blood counts. Such treatment consists of giving red blood cell transfusions to correct anemia, platelet transfusions to treat or prevent serious bleeding, and antibiotics to treat or prevent infections. Supportive care is a critical component in the management of aplastic anemia, aiming to address symptoms, complications, and overall patient well-being. Patients often require regular blood transfusions to alleviate anemia and manage low platelet counts.

Aplastic Anemia Epidemiology

As the market is derived using a patient-based model, the aplastic anemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total incident cases of aplastic anemia, severity-specific cases of aplastic anemia, and age-specific cases of aplastic anemia in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2020 to 2034.

• The total incident cases of aplastic anemia in the 7MM comprised approximately ~2,500 cases in 2023 and are projected to increase during the forecast period.
• Among the 7MM, the Japan accounted for the highest number of incident cases of aplastic anemia, i.e., ~800 cases in 2023.
• According to estimates, aplastic anemia is more common in the older population; the highest incident cases were in age group of =60 years, accounting for ~60% cases, and ~40% were < 60 in 2020 in the 7MM.
• In 2023, Among EU4 and the UK, Germany accounted for the largest number of aplastic anemia cases, whereas Spain occupied the bottom of the ladder.

Aplastic Anemia Drug Chapters

The drug chapter segment of the aplastic anemia report encloses a detailed analysis of aplastic anemia marketed drugs and emerging pipeline drugs. It also helps understand the aplastic anemia pivotal clinical trial details, recent and expected market approvals, patent details, each drug's advantages and disadvantages, the latest news, and recent deals and collaborations.

Marketed Drug

PROMACTA/REVOLADE (eltrombopag): Novartis

PROMACTA/REVOLADE (eltrombopag) tablets contain eltrombopag olamine, a small molecule thrombopoietin (TPO) receptor agonist for oral administration. Eltrombopag, sold under the brand name PROMACTA, among others, is a medication used to treat thrombocytopenia and severe aplastic anemia (SAA). Eltrombopag is sold under the brand name REVOLADE outside the US and is marketed by Novartis. In August 2014, the US FDA approved a sNDA for the once-daily use of PROMACTA in patients with severe aplastic anemia (SAA) who had an insufficient response to immunosuppressive therapy. And later in November 2018, the us FDA expanded the label for PROMACTA (eltrombopag) to include first-line treatment for adults and pediatric patients 2 years and older with SAA in combination with IST.

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. It acts directly on megakaryocytic progenitor cells and exerts platelet hematopoietic effects.

Emerging Drug

REGN7257: Regeneron Pharmaceuticals

REGN7257 is 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. The drug REGN7257 is presently undergoing evaluation in a Phase I/II clinical trial to assess its safety and tolerability in patients with severe aplastic anemia (SAA) who have either relapsed after immunosuppressive therapy (IST) or are refractory to IST.

OMISIRGE (omidubicel): Gamida Cell

OMISIRGE (omidubicel-onlv) is a cryopreserved nicotinamide-modified allogeneic hematopoietic progenitor cell therapy derived from cord blood and manufactured utilizing a proprietary NAM-based technology producing enriched HPCs. 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. 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.

Aplastic Anemia Market Outlook

In real-world scenarios, the approach to treating NSAA differs significantly from SAA/VSAA. Unlike SAA/VSAA, hematopoietic stem cell transplantation (HSCT) is not employed in NSAA cases. The criteria for determining when to initiate treatment for NSAA are less clearly defined, and there is a lack of comprehensive recommendations for clinical evaluation and therapeutic strategies. The ultimate management of NSAA predominantly relies on the expertise of individual medical centers. 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. In the realm of second-line treatments, patients with aplastic anemia are considered eligible for transplantation in cases of refractory status following first-line IST. IST in the form of horse ATG + CyA has been extensively researched regimen in Europe, Asia, and the United States over several decades. Recent endeavors to enhance the efficacy of horse ATG have involved augmenting immunosuppression with agents such as mycophenolate, sirolimus, rabbit ATG, alemtuzumab, and cyclophosphamide.

Approximately a decade ago, notable progress was made with the approval of thrombopoietin receptor agonists for immune thrombocytopenia, effectively inducing platelet count recovery in refractory cases. Distinguishing itself from erythropoietin and G-CSF, the Tpo receptor is expressed in stem cells. Tpo receptor knockout models revealed significant reductions not only in megakaryocytes and platelets but also in HSCs. In 2014, the US FDA approved PROMACTA for the treatment of aplastic anemia, which then approved in Europe and Japan in 2015 and 2017, respectively, under the brand name REVOLADE. Later, another Tpo receptor agonist, ROMIPLATE, was granted approval in Japan in June 2019 for aplastic anemia cases where conventional treatments have proven insufficient.

With respect to the emerging pipeline, there is a scarcity of advancements in SAA treatment, with only two companies in the 7MM actively engaged in developing therapies - Regeneron Pharmaceuticals with REGN7257 and Gamida Cell with omidubicel. This underscores the crucial need for additional research and development to broaden the spectrum of treatment options for this complex condition.

Key Findings

As per the analyst's estimates, the potential drugs that can mark a significant change in the forecast period include xx, xx, and others.

• The total market size of aplastic anemia in the 7MM is approximately USD ~270 million in 2023 and is projected to increase during the forecast period (2024-2034).
• Among EU4 and the UK, Germany accounted for the maximum market size in 2023, while the UK occupies the bottom of the ladder.
• Among the therapies, PROMACTA is expected to generate the highest revenue in the 7MM by 2034.

Aplastic Anemia Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020-2034. A medium uptake is expected by REGN7257.

Aplastic Anemia Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III and Phase II. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for aplastic anemia emerging therapy.

KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts contacted for insights on aplastic anemia evolving treatment landscape, patient reliance on conventional therapies, patient’s therapy switching acceptability, and drug uptake, along with challenges related to accessibility, include Medical/scientific writers; Professors; MD, FACS, Head of the Departments, Universities, and others.

The analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Centers such as the East Tennessee State University, Düsseldorf Clinics Association, Duke University School of Medicine, etc. were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or aplastic anemia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

The analyst analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry.

In efficacy, the trial’s primary and secondary outcome measures are evaluated. Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials.

Market Access and Reimbursement

Reimbursement is a crucial factor affecting the drug’s market access. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, payers and other industry insiders are considering many payment models.

Patients prescribed PROMCATA might be eligible to receive a free 14-day supply by mail while beginning therapy. Additionally, Novartis also provides eligible patients with a temporary supply of PROMACTA through its Voucher Program. Through its Universal Copay Card, eligible patients with private insurance may pay USD 0 per month, with Novartis paying the remaining copay up to USD 15,000 per calendar year. Patients facing financial hardship and/or having no third-party insurance coverage might be eligible to receive PROMACTA for free through the Novartis Patient Assistance Foundation. About 99% of the patients being prescribed PROMACTA have it included in their health coverage, with more than 70% of Medicare and commercially insured patients paying less than USD 10,000 in out-of-pocket costs.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, descriptive overview of Aplastic Anemia, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the aplastic anemia market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM aplastic anemia market.

Aplastic Anemia Report Insights

• Patient Population
• Therapeutic Approaches
• Aplastic Anemia Pipeline Analysis
• Aplastic Anemia Market Size and Trends
• Existing and future Market Opportunity

Aplastic Anemia Report Key Strengths

• Eleven Years Forecast
• The 7MM Coverage
• Aplastic Anemia Epidemiology Segmentation
• Key Cross Competition
• Drugs Uptake and Key Market Forecast Assumptions

Aplastic Anemia Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

• What was the aplastic anemia total market size, the market size by therapies, and market share (%) distribution in 2020, and what would it look like in 2034? What are the contributing factors for this growth?
• Which class is going to be the largest contributor in 2034?
• What will be the market size of PROMACTA in 2025?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
• What are the disease risks, burdens, and unmet needs of aplastic anemia? What will be the growth opportunities across the 7MM concerning the patient population of aplastic anemia?
• What is the historical and forecasted aplastic anemia patient pool in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• What are the current options for the treatment of aplastic anemia? What are the current guidelines for treating aplastic anemia in the US and Europe?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies?
• What key designations have been granted for the emerging therapies for aplastic anemia?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?

Reasons to Buy

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the aplastic anemia market.
• Insights on patient burden/disease incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the Analyst view section to provide visibility around leading classes.
• Highlights of Access and Reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.