Diamond Blackfan Anemia Market: Epidemiology, Therapies, Companies, DelveInsight | AstraZeneca, Novartis AG, Pfizer Inc., Sanofi, Merck KGaA

Diamond Blackfan Anemia therapies are expected to boost the Diamond Blackfan Anemia Market in the upcoming years.

DelveInsight has launched a new report on "Diamond Blackfan Anemia - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Diamond Blackfan Anemia, historical and forecasted epidemiology as well as the Diamond Blackfan Anemia market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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Some of the key facts of the Diamond Blackfan Anemia Market Report:

The United States holds the largest share of the Diamond Blackfan Anemia (DBA) market, significantly outpacing other regions including the EU4 (Germany, France, Italy, Spain), the UK, and Japan. Key players operating in this space include AstraZeneca, Novartis AG, Pfizer Inc., Sanofi, Merck KGaA, among others.
DBA is a rare inherited bone marrow failure disorder that impairs red blood cell production, often presenting in infancy or early childhood. Clinical features include anemia, pale skin, stunted growth, fatigue, and congenital anomalies such as thumb malformations and cardiac defects.
In 2023, approximately 7,870 diagnosed DBA cases were reported across the seven major markets (7MM), with the U.S. accounting for nearly 70% of these. The prevalence of DBA is expected to grow at a compound annual growth rate (CAGR) over the forecast period (2020-2034).
St. Jude has advanced a lentiviral-based gene therapy targeting RPS19 mutations, reaching promising preclinical safety and efficacy data. This approach may allow low-intensity conditioning prior to autologous stem-cell transplant, reducing toxicity. The therapy is progressing toward a clinical trial planned for 2026 .
Among European countries, Germany reported the highest number of DBA cases, followed by the UK, which represented 20% of diagnosed prevalence across the EU4 and UK combined.
In the U.S., around 46% of diagnosed DBA cases in 2023 were associated with mutations in the RPS19 gene, underscoring its key role in ribosomal protein function. Craniofacial and musculoskeletal anomalies were the most frequently observed congenital features, with craniofacial anomalies present in over 30% of patients across the 7MM in 2023.
Despite its burden, no FDA-approved therapies currently exist for DBA. Treatment approaches include corticosteroids, chronic blood transfusions, iron chelation, and potentially curative hematopoietic stem cell transplantation (HSCT). In 2023, corticosteroids led the first-line treatment market, generating $1.1 million in revenue, ahead of other therapies.
In 2023, an estimated 7,870 diagnosed cases of Diamond Blackfan anemia (DBA) were recorded across the seven major markets (7MM), with the United States accounting for nearly 70% of the total. The prevalence of DBA is expected to rise at a compound annual growth rate (CAGR) through 2034.
Among the EU4 countries and the UK, Germany reported the highest number of DBA cases in 2023, followed by the UK, which contributed around 20% of the diagnosed cases in this regional group.
Key Diamond Blackfan Anemia companies such as AstraZeneca, Novartis AG, Pfizer Inc., Sanofi, Merck KGaA, and others are evaluating new drugs for Diamond Blackfan Anemia to improve the treatment landscape.

Diamond Blackfan Anemia Overview

Diamond Blackfan Anemia (DBA) is a rare genetic disorder characterized by bone marrow failure that specifically impairs red blood cell production. Typically identified in infancy or early childhood, patients often present with severe anemia, fatigue, pale skin, and growth delays. Around 50% also show physical abnormalities, including thumb defects, facial deformities, or heart issues.
DBA is primarily caused by mutations in ribosomal protein genes-most commonly RPS19-which are essential for protein synthesis. These genetic mutations disrupt red blood cell development, and while many cases are inherited in an autosomal dominant pattern, some arise spontaneously.

Diagnosis involves clinical evaluation, blood tests showing macrocytic anemia with relatively normal white cells and platelets, elevated erythrocyte adenosine deaminase (eADA) levels, and bone marrow biopsy showing reduced red cell precursors. Genetic testing confirms the diagnosis and guides treatment. Early detection is key to improving outcomes and preventing complications.
Diamond Blackfan Anemia Market Outlook

The current treatment landscape for Diamond Blackfan anemia (DBA) primarily includes glucocorticosteroids, red blood cell transfusions, and hematopoietic stem cell transplantation (HSCT). However, each approach has significant limitations, including steroid resistance, iron overload from transfusions, and the risks associated with transplantation.

In 2023, the total DBA market size across the seven major markets (7MM: the U.S., EU4, UK, and Japan) was valued at USD 1.56 million, with projections indicating steady growth through 2034. The United States dominates this market, driven by its larger patient population and greater scope for therapeutic innovation.

Within Europe, Germany held the largest market share among the EU4 and the UK, with an estimated value of USD 0.06 million in 2023. The UK followed, while XX represented the smallest market within this group.

Currently, there are no FDA-approved therapies specifically for DBA, and few pipeline treatments are being developed. This presents a significant opportunity for novel therapies, such as gene-based approaches, to address unmet needs and transform patient outcomes.

In 2023, corticosteroids remained the leading therapy in the DBA market, generating USD 1.1 million in revenue-outperforming red blood cell transfusions and HSCT as the preferred first-line treatment.

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Scope of the Diamond Blackfan Anemia Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Diamond Blackfan Anemia Companies: AstraZeneca, Novartis AG, Pfizer Inc., Sanofi, Merck KGaA, and others
Diamond Blackfan Anemia Therapeutic Assessment: Diamond Blackfan Anemia current marketed and Diamond Blackfan Anemia emerging therapies
Diamond Blackfan Anemia Market Dynamics: Diamond Blackfan Anemia market drivers and Diamond Blackfan Anemia market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
Diamond Blackfan Anemia Unmet Needs, KOL's views, Analyst's views, Diamond Blackfan Anemia Market Access and Reimbursement

To know what's more in our Diamond Blackfan Anemia report, visit https://www.delveinsight.com/report-store/diamond-blackfan-anemia-dba-aase-syndrome-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Key benefits of the Diamond Blackfan Anemia Market Report:
The Diamond Blackfan Anemia market report covers a descriptive overview and comprehensive insight of the Diamond Blackfan Anemia Epidemiology and Diamond Blackfan Anemia market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
The Diamond Blackfan Anemia market report provides insights into the current and emerging therapies.
The Diamond Blackfan Anemia market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Diamond Blackfan Anemia market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Diamond Blackfan Anemia market.

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Table of Contents

1. Report Introduction
2. Executive Summary
3. SWOT analysis
4. Diamond Blackfan Anemia Patient Share (%) Overview at a Glance
5. Diamond Blackfan Anemia Market Overview at a Glance
6. Diamond Blackfan Anemia Disease Background and Overview
7. Diamond Blackfan Anemia Epidemiology and Patient Population
8. Country-Specific Patient Population of Diamond Blackfan Anemia
9. Diamond Blackfan Anemia Current Treatment and Medical Practices
10. Unmet Needs
11. Diamond Blackfan Anemia Emerging Therapies
12. Diamond Blackfan Anemia Market Outlook
13. Country-Wise Diamond Blackfan Anemia Market Analysis (2020-2034)
14. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Diamond Blackfan Anemia Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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Related Reports:

Diamond Blackfan Anemia Pipeline Insights, DelveInsight

"Diamond Blackfan Anemia Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Diamond Blackfan Anemia market. A detailed picture of the Diamond Blackfan Anemia pipeline landscape is provided, which includes the disease overview and Diamond Blackfan Anemia treatment guidelines.

Contact Info:

Kritika Rehani, Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
www.delveinsight.com
+14699457679

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.