Dublin, Jan. 02, 2025 (GLOBE NEWSWIRE) -- The "Diamond-Blackfan Anemia - Market Insight, Epidemiology and Market Forecast - 2034" report has been added to ResearchAndMarkets.com's offering.
This report delivers an in-depth understanding of the indication Diamond Blackfan Anemia, historical and forecasted epidemiology as well as the Diamond Blackfan Anemia market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
The Diamond Blackfan Anemia market report provides real-world prescription pattern analysis, approved drugs, market share of individual therapies, and historical and forecasted 7MM Diamond Blackfan Anemia market size from 2020 to 2034. The report also covers current Diamond Blackfan Anemia treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's underlying potential.
Key Highlights
- Diamond-Blackfan anemia (DBA) is a rare genetic disorder that primarily affects the bone marrow's ability to produce red blood cells, leading to severe anemia. It is usually diagnosed in infancy or early childhood, with symptoms like pale skin, fatigue, poor growth, and physical abnormalities such as thumb deformities or heart defects in some cases.
- In 2023, approximately 7,870 diagnosed cases of Diamond-Blackfan Anemia were reported across the seven major markets (7MM), with the United States representing about 70% of these cases.
- In 2023, Germany reported the highest number of Diamond-Blackfan Anemia cases among the EU4 countries and the UK, followed by the UK, which accounted for 20% of the total diagnosed prevalent cases in these regions.
- In the United States, a substantial proportion of Diamond-Blackfan Anemia cases reported in 2023, approximately 46%, were associated with mutations in the RPS19 gene. This high prevalence is linked to the gene's significant role in ribosomal protein synthesis.
- Several congenital anomalies are commonly associated with Diamond-Blackfan Anemia, with craniofacial and musculoskeletal anomalies being the most prevalent. Notably, craniofacial anomalies were observed in over 30% of DBA patients across the 7MM in the year 2023.
- Despite the significant impact of DBA on the patients' health, no FDA-approved treatments currently exist for the condition. Management primarily involves the use of corticosteroids combinations, chronic blood transfusions, iron chelation, and potentially curative hematopoietic stem cell transplantation (HSCT). In 2023, corticosteroids dominated the therapeutic landscape for DBA, generating USD 1.1 million in revenue, surpassing other treatment options for first line of therapy.
Diamond Blackfan Anemia Epidemiology
The Diamond Blackfan Anemia epidemiology chapter in the report provides historical as well as forecasted incidence in the 7MM covering the United States, EU4 countries, the United Kingdom, and Japan from 2024 to 2034.
The Diamond Blackfan Anemia epidemiology is segmented with detailed insights into:
- Total Diagnosed Prevalent Cases of Diamond Blackfan Anemia in the 7MM [2020-2034]
- Cases of DBA by Mutated Gene in the 7MM [2020-2034].
- Cases of DBA by Congenital Anomaly in the 7MM [2020-2034]
- Total Treated Cases of DBA in the 7MM [2020-2034]
Key Epidemiological Highlights
- In 2023, approximately 7,870 diagnosed cases of Diamond-Blackfan Anemia were reported across the seven major markets (7MM), with the United States representing about 70% of these cases.
- In 2023, Germany reported the highest number of Diamond-Blackfan Anemia cases among the EU4 countries and the UK, followed by the UK, which accounted for 20% of the total diagnosed prevalent cases in these regions.
- In the United States, a substantial proportion of Diamond-Blackfan Anemia cases reported in 2023, approximately 46%, were associated with mutations in the RPS19 gene. This high prevalence is linked to the gene's significant role in ribosomal protein synthesis.
- Several congenital anomalies are commonly associated with Diamond-Blackfan Anemia, with craniofacial and musculoskeletal anomalies being the most prevalent. Notably, craniofacial anomalies are observed in over 30% of DBA patients across the seven major markets (7MM).
- In 2023, 50% of Diamond-Blackfan Anemia patients in the United States advanced to second-line treatments after initially receiving first-line therapy indicating corticosteroids may become less effective over time.
Diamond Blackfan Anemia Market Outlook
Diamond-Blackfan Anemia (DBA) current treatment landscape includes glucocorticosteroids, blood transfusions, and hematopoietic stem cell transplantation (HSCT). However, each of these therapies faces limitations: steroid resistance, iron overload, and transplantation risks. The United States leads the 7MM (United States, Europe, and Japan) market for Diamond-Blackfan Anemia, driven by its large patient population and opportunities for innovation.
- The United States represents the largest share of the Diamond Blackfan Anemia market, accounting for a significant proportion compared to the EU4, the United Kingdom, and Japan.
- Among EU4 and the UK, the Germany had the largest market size accounting for approximately USD 0.06 million, followed by the United Kingdom
- As no FDA-approved therapies currently exist and no emerging players are addressing this gap, there is a substantial opportunity for new, effective treatments, such as gene therapy, to make a significant impact and meet the needs of DBA patients.
- In 2023, corticosteroids dominated the therapeutic landscape for DBA, generating USD 1.1 million in revenue, surpassing other treatment options such as red blood cell transfusions and hematopoietic stem cell transplantation.
Scope of the Report
- The report covers a segment of key events, an executive summary, descriptive overview of Diamond Blackfan Anemia, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
- Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, and disease progression along with country specific treatment guidelines.
- Additionally, an all-inclusive account of the current therapies, along with the elaborative profiles of prominent therapies and how will they have an impact on the current treatment landscape.
- A detailed review of the Diamond Blackfan 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 in shaping and driving the 7MM Diamond Blackfan Anemia.
Diamond Blackfan Anemia Report Insights
- Patient Population
- Therapeutic Approaches
- Diamond Blackfan Anemia Market Size and Trends
- Existing and future Market Opportunity
Diamond Blackfan Anemia Report Key Strengths
- Eleven Years Forecast
- 7MM Coverage
- Diamond Blackfan Anemia Epidemiology Segmentation
- Inclusion of Country specific treatment guidelines
- KOL's feedback on approved therapies
- Conjoint analysis
- Drugs Uptake and Key Market Forecast Assumptions
For more information about this report visit https://www.researchandmarkets.com/r/lnm9v2
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