The Changing Market Dynamics of Immune Thrombocytopenia, Aplastic Anemia, and Diamond-Blackfan Anemia | DelveInsight Insights

Blood disorders affect millions worldwide—an estimated 5% of the global population suffers from conditions like anemia, and others. Anemia is a silent epidemic, with over 2 billion people affected globally, often caused by iron deficiency or chronic diseases. With ongoing research, the treatment landscape for blood disorders continues to evolve, from targeted therapies to life-saving gene editing technologies.

LAS VEGAS, Dec. 9, 2024 /PRNewswire/ -- Blood disorders are a group of conditions that affect the production, function, or structure of blood cells, leading to various health complications. These disorders can involve issues with red blood cells, white blood cells, platelets, or the plasma components of blood. Common types of blood disorders include anemia, where there is a deficiency of red blood cells or hemoglobin, leading to fatigue and weakness, and thrombocytopenia, characterized by low platelet counts, which can cause excessive bleeding. The causes of blood disorders range from genetic factors and autoimmune conditions to infections and environmental factors. Treatment options depend on the specific disorder and may include medications, blood transfusions, or bone marrow transplants.

DelveInsight has recently released a series of epidemiology-based market reports focusing on blood disorders including Immune Thrombocytopenia, Aplastic Anemia, and Diamond-Blackfan Anemia. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034  segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Additionally, the reports feature an examination of prominent companies working with their lead candidates in different stages of clinical development. Let's deep dive into the assessment of these blood disorders markets individually.

Immune Thrombocytopenia Market

Immune thrombocytopenic purpura (ITP) is an autoimmune condition marked by a low platelet count, resulting in bruising and bleeding. It manifests as acute in children and chronic in adults. The primary treatments are corticosteroids, intravenous immunoglobulin (IVIg), and rituximab. 

In the 7MM, the United States reported the highest prevalence of ITP, with nearly 66,500 cases in 2023. Primary ITP, which accounts for 80% of cases, occurs without any underlying diseases, while secondary ITP, making up about 20% of cases, is linked to conditions such as systemic lupus erythematosus (SLE), medications, infections, and other autoimmune diseases.

In immune thrombocytopenia, the initial treatment generally consists of corticosteroids, with prednisone prescribed at 1 mg/kg/day for 21-28 days, followed by a gradual taper. High-dose dexamethasone (40 mg/day for 4 days) may lead to a more pronounced platelet response. Intravenous immunoglobulin (IVIG) or intravenous anti-D (Rho[D] immune globulin) may be used as first-line options, particularly in urgent situations requiring quick platelet increases.

However, many adults experience relapses or do not respond to first-line therapy, necessitating second-line treatments. IVIG is effective in 70-80% of cases, and anti-D works in 50-70% of RhD-positive patients. Splenectomy is a well-established second-line option, though it carries risks such as infection and bleeding.

Rituximab, a monoclonal antibody that targets CD20, has shown strong efficacy both before and after splenectomy. Thrombopoietin-receptor agonists, such as romiplostim and eltrombopag, are also effective second-line treatments as they promote platelet production by activating the thrombopoietin receptor.

In the US, the approved treatments for ITP are DOPTELET, TAVALISSE, PROMACTA, and NPLATE. The EU market for thrombocytopenia is primarily driven by both approved and off-label therapies. In the EU, approved treatments include DOPTELET (avatrombopag), TAVALISSE/TAVLESSE (fostamatinib), PROMACTA/REVOLADE (eltrombopag), and NPLATE/Romiplate (romiplostim). RITUXAN is only approved in Japan for the treatment of ITP.

Learn more about the FDA-approved drugs for ITP @ Drugs for Immune Thrombocytopenic Purpura Treatment

As per DelveInsight analysis, in 2023, the total ITP market size in the 7MM was USD 3.1 billion. As per the estimates, PROMACTA (eltrombopag) achieved the highest revenue among all medications in the 7MM in 2023. The market is expected to show positive growth, mainly attributed to the increasing cases and also, the launch of upcoming therapies during the forecast period (2024–2034).

Immune Thrombocytopenic Purpura Pipeline Therapies and Key Companies

• Rilzabrutinib (PRN-1008): Sanofi/Principia Biopharma
• Mezagitamab (TAK-079): Millennium Pharmaceuticals/Takeda
• Efgartigimod (ARGX-113): argenx
• BT-595: Biotest

Dive deeper for rich insights into the Immune Thrombocytopenic Purpura Clinical Trials

Aplastic Anemia Market

Aplastic anemia is a rare and serious non-cancerous condition marked by the autoimmune destruction of early blood-forming cells. The global incidence varies from 0.7 to 7.4 cases per million people annually, with higher rates observed in Asia compared to Europe and the United States.

As per DelveInsight estimates, the total Incident population of aplastic anemia in the 7MM were around 2,500 cases in 2023. These cases are projected to increase during the forecast period. Based on severity, severe and very severe aplastic anemia patients contributed roughly 65%-80% of the total aplastic anemia patient population.

HSCT remains the standard treatment for patients under 40 years old, while Immunosuppressive Therapies (IST) are the preferred approach for patients aged 40 and above. In terms of pharmacological treatments, Novartis' PROMACTA/REVOLADE is currently the leading revenue driver, followed by ISTs across the 7MM. 

According to Novartis, the primary revenue drivers for PROMACTA/REVOLADE are its use in patients with chronic immune thrombocytopenic purpura (ITP) and severe aplastic anemia (SAA). PROMACTA was first approved in 2014 for use in relapsed/refractory SAA patients in the United States, with approvals following in Europe in 2015 and Japan in 2017. However, it was approved for use as a first-line treatment in combination with ISTs like ATG in the US in 2018, which expanded treatment options for first-line patients and contributed to increased revenue.

The total aplastic anemia market size was found to be ~USD 270 million in 2023 in the 7MM, which is further expected to increase by 2034 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. 

Aplastic Anemia Pipeline Therapies and Key Companies

• REGN7257: Regeneron Pharmaceuticals
• OMISIRGE (omidubicel): Gamida Cell

For a comprehensive view of the aplastic anemia market, check out the Aplastic Anemia Market Assessment

Diamond-Blackfan Anemia Market

Diamond-Blackfan anemia (DBA) is a rare genetic condition that primarily disrupts the bone marrow's ability to produce red blood cells, resulting in severe anemia. It is typically diagnosed during infancy or early childhood and presents with symptoms such as pale skin, fatigue, stunted growth, and physical abnormalities, which may include thumb deformities or heart defects in certain cases. 

In 2023, there were around 7,870 reported cases of Diamond-Blackfan anemia across the 7MM, with the United States accounting for about 70% of these cases. Within the US, approximately 46% of cases in 2023 were linked to mutations in the RPS19 gene, which plays a crucial role in ribosomal protein synthesis, contributing to its high prevalence in DBA.

Diamond-Blackfan anemia is frequently associated with several congenital anomalies, with craniofacial and musculoskeletal defects being the most common. Craniofacial abnormalities were noted in over 30% of DBA patients across the 7MM in 2023. 

Although DBA has a significant impact on patient health, there are currently no FDA-approved treatments available for the condition. Treatment primarily involves corticosteroid combinations, chronic blood transfusions, iron chelation, and potentially curative hematopoietic stem cell transplantation (HSCT). 

In 2023, the total market size for Diamond-Blackfan anemia was USD 1.56 million in the 7MM and is projected to increase by 2034. The United States represents the largest share of the Diamond Blackfan Anemia market, accounting for a significant proportion compared to the EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

In 2023, corticosteroids were the leading therapy for DBA, generating USD 1.1 million in revenue, making them the preferred first-line treatment option. Since there are no FDA-approved therapies available and no emerging companies are working to fill this gap, there is a significant opportunity for innovative treatments, like gene therapy, to make a meaningful difference and address the needs of DBA patients.

For a deeper understanding of the Diamond-Blackfan anemia market landscape, explore the Diamond-Blackfan Anemia Market Outlook

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SOURCE DelveInsight Business Research, LLP