Diamond-Blackfan Anemia (DBAAASE) Syndrome Therapeutics Market Snapshot (2023 to 2033)

The global diamond-blackfan anemia (DBAAASE) syndrome therapeutics market size is expected to surpass an impressive valuation of USD 4.8 billion in 2023 and is projected to exhibit a CAGR of 4.2% from 2023 to 2033. A valuation of USD 7.29 billion is expected for the market by the end of the aforementioned period of assessment

Diamond-blackfan anemia is an inherited blood disorder that impairs bone marrow's capacity to generate red blood cells. It is a form of bone marrow malfunction. It is also referred to as obtained pure red cell aplasia and is induced by genetic mutations. Light skin, loss of energy, fast heartbeat, unusual thumbs, low bone density, cleft palate, and heart and kidney difficulties are all symptoms of this disorder. Erythrogenesis imperfecta, DBA, congenital hypoplastic anemia, inherited erythroblastopenia, and congenital pure red cell aplasia is all identities for this condition.

Although most cases are isolated, approximately 45% of people with Diamond-Blackfan anemia inherit the disorder from one of their parents. The increased incidences of diamond-blackfan anemia, burgeoning healthcare expenditure, advancement of therapeutic approaches, soaring federal funding, and surging strategies by government and private organizations to raise illnesses awareness are the aspects that will drive the diamond-blackfan anemia treatment market.

Report Attribute Details
Expected Market Value (2023) USD 4.8 billion
Anticipated Forecast Value (2033) USD 7.29 billion
Projected Growth Rate (2023 to 2033) 4.2% CAGR

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2018 to 2022 Diamond–Blackfan Anemia (DBAAASE) Syndrome Therapeutics Demand Analysis vs. Forecast 2023 to 2033

Since hematologic complications develop in 90% of infected persons during their first year of life, a rise in the number of blood disorders propelled the global diamond-blackfan anemia treatment market over the historical period. Aplastic anemia is being raised awareness by organizations such as the National Organization for Rare Disorders, Inc. and CancerIndex, among others. The Aplastic Anemia Trust's spring awareness campaign was launched in February 2019.

The pandemic has diminished the number of charitable blood donations and interrupted the global supply chain for medical equipment. Reduced donor inflow, a lack of staff members, a scarcity of blood units, and general concern about transfusion-transmitted COVID-19 were among the major obstacles confronted by blood banks and clinics from 2020 to 2021. From 2018 to 2022, the market witnessed significant growth, registering a CAGR of 3.8%.

Furthermore, rising government support for R&D, changing lifestyles, and rapidly evolving technology are expected to drive market expansion over the forecast period.

Which are Some Prominent Drivers of the DBAAASE Syndrome Therapeutics Market?

Supportive Laws by Government Entities to Spur Market Expansion

The existence of supportive regulations by governmental agencies, such as the Orphan Drug Act, which encourages product development by awarding orphan drug certification to potential drug delivery developed by drug manufacturers, is anticipated to boost industry growth. This initiative promotes study and advancement in the treatment of diamond-blackfan anemia.

According to a recent article, the US Food and Drug Administration has approved approximately 21 to 22 novel drugs and biopharmaceuticals for more than 800 rare disease indications. Furthermore, burgeoning genomics investigations have resulted in the discovery of effective biologic drugs for rare genetic disorders. These aspects have significantly boosted the market for diamond-blackfan anemia treatment.

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Federal Spending on Research, Development and Medical Approaches to drive Market Growth

Increasing government expenditure on research, development, and therapeutic interventions is expected to propel the market's growth in the coming decade. For instance, in India, the National Policy for Rare Diseases, 2021 (the Policy) has been accomplished and publicly disclosed.

According to the provisions envisioned in the National Policy for Rare Diseases, 2021, concerning providing economic assistance to individuals with rare diseases, it is proposed that funding will be offered under the umbrella scheme of RAN, i.e. an amount up to Rs. 20 lakhs (USD 24,358) only for privileged claimants will be offered by the Central Government. As a result, the market for diamond-blackfan anemias treatment is expected to grow.

What are the Challenges Projected to be Faced by the Market?

High Costs and Complicated Side Effects to Hinder Market Growth

The elevated cost of treatment and complications linked to the disease, such as leukemia, osteosarcoma, acute myeloid leukemia, and myelodysplastic syndrome, will impede overall growth and pose significant challenges to the diamond-blackfan anemia treatment market in the coming years. A stem cell transplant costs more than USD 290,000. As a consequence, the elevated cost of treatment is inclined to stifle growth in the market.

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Region-Wise Insights

What Makes North America the Largest Market of DBAAASE Syndrome Therapeutics?

Major notable stakeholders, high disposable income to drive the North American market

Owing to the existence of major prominent stakeholders, high expendable income, elevated health expenditures, and a well-established medical industry in this region, North America dominates the diamond-blackfan anemia treatment market. According to a report released by Clevel and Clinic, the DBA Registry of North America, a database with data on individuals who have this disorder, 25% of people with DBA die by the age of 50 from associated medical disorders.

North America will dominate the market in 2021 with a sales revenue of more than 60.0% owing to the high incidence of diseases, sophisticated healthcare infrastructure, and unique product authorizations for diagnosis. Tezepelumab received orphan drug certification from the US FDA in October 2021. Exposure to orphan drugs for diamond-blackfan anemia treatment may enhance patient adherence, thereby broadening the consumer base and elevating industry revenue. North America is expected to hold approximately 40% market share.

Why is Europe Considered a Lucrative Region for the DBAAASE Syndrome Therapeutics Market?

Innovative drugs development to drive the European market

Autologous CD34+ enriched cells transduced with a self-inactivating lentiviral vector containing the codon-optimized RPS19 gene drug were identified as an orphan medication for the treatment of Diamond-Blackfan anemia in the European Union on November 12, 2021. This means that the developer will receive scientific and regulatory assistance from the EMA to advance their medicine to the point where they can apply for marketing authorization, which will boost the market growth in this region.

DBA UK is a registered charity (1083179) ruled by a written constitution and operated by elected volunteer groups. They provide support, studies, and hope to the DBA community by introducing families together to converse about their experiences, convey the most up-to-date medical records, and raise funds to help those with DBA in the United Kingdom. As a result, this venture is predicted to benefit the European market. The Europe market is expected to expand with a CAGR of 3.7%.

Category-Wise Insights

Which Treatment Segment is Expected to Witness High Demand?

Corticosteroids are among the most widely prescribed Medicine Classes Worldwide

Customers are drawn to corticosteroids because they are available in a variety of forms, including oral capsules or tablets, oral syrups, injectables, topical creams or gels, and nasal sprays.

Corticosteroids are also one of the most frequently prescribed drug classes globally, with a market worth more than USD 10 billion per year, according to a National Library of Medicine report. As a result of the aforementioned factors, the corticosteroids segment is expected to grow significantly during the forecast period.

What is another Treatment Segment Expected to Grow?

The blood Transfusions Segment is predicted to Expand Quickly

The adoption of advanced molecular systems by market leaders accelerates the transition from test cases and instruments to automated devices. These automated systems have been critical in detecting Transfusion Transmissible Infections (TTIs) in samples taken and can accurately avoid human error during the transfusion method. As a result, the blood transfusions segment is expected to expand rapidly, with a CAGR of 6.9%.

Start-ups Specializing in DBAAASE Syndrome Therapeutics

Prominent start-up players in the anorectal malformation treatment sector are introducing novel and innovative product lines to strengthen their market presence, and the aforementioned start-ups have left no stone unturned. The following are some specific examples of significant t start-ups:

  • EryPharm is a startup that was founded in 2016. EryPharm entered the market in April 2021 with a sophisticated tech that can generate red blood cells in huge volumes and build innovative blood transfusion sources. The company intends to gain entry into a pilot production phase and expand the production of Cultured Red Blood Cells (cRBC) using Hematopoietic Stem Cells (HSC).
  • In 2017, Torax Biosciences Limited was established. Torax Biosciences Limited introduced its B2B IVD bulk reagents for latex serology and blood grouping assays to the industry in February 2019. The company intends to supply its distribution network, production facilities, and clinics with visual latex agglutination assay ASO, CRP, and RF, as well as blood grouping assays in FFMU or packed formats.

Market Competition

Key players in the Diamond-Blackfan Anemia (DBAAASE) Syndrome Therapeutics market are Sumitomo Corporation, Johnson & Johnson Private Limited, Novartis AG, Pfizer Inc., Sanofi, Merck KGaA, LEO Pharma A/S, Cipla Inc., GlaxoSmithKline plc, AstraZeneca, Abbott Laboratories and Bayer AG.

  • AstraZeneca Plc. acquired Alexion Pharmaceuticals, a global biotech company, in July 2021. With this acquisition, the company will be able to enter the market for medicines for rare diseases, marking the start of a new chapter. Furthermore, the acquisition strengthened AstraZeneca's scientific presence in immunology and would allow the company to proceed to pioneer the discovery and creation of medicines for rare disease patients through Alexion's new complement-biology system and sturdy pipeline.
  • Pfizer Inc. collaborated with Sirana Pharma, a biotechnology company, in June 2022. The collaboration would aid Pfizer in the recognition and affirmation of an innovative treatment notion for rare and severe bone diseases. In addition, the procurement would initially involve the adoption of Sirana's proprietary microRNA approach, which aims for significant restorative recovery of diseased muscle and bone tissue.

Report Scope

Report Attribute Details
Expected Market Value (2023) USS 4.8 billion
Anticipated Forecast Value (2033) USD 7.29 billion
Projected Growth Rate (2023 to 2033) 4.1% CAGR
Base Year for Estimation 2022
Historical Data 2018 to 2022
Forecast Period 2023 to 2033
Quantitative Units Revenue in USD Billion and CAGR from 2023 to 2033
Report Coverage Revenue Forecast, Volume Forecast, Company Ranking, Competitive Landscape, Growth Factors, Trends, and Pricing Analysis
Segments Covered Treatment, End User, Region
Regions Covered North America; Latin America; Europe; Asia Pacific; Middle East & Africa (MEA)
Key Countries Profiled USA, Canada, Brazil, Mexico, Germany, UK, France, Spain, Italy, China, Japan, South Korea, Singapore, Thailand, Indonesia, Australia, New Zealand, GCC Countries, South Africa, Israel
Key Companies Profiled Sumitomo Corporation; Johnson & Johnson Private Limited; Novartis AG; Pfizer Inc.; Sanofi S.A; Merck & Co. Inc.; LEO Pharma A/S; Cipla Limited; GSK Plc.; AstraZeneca Plc.; Abbott Laboratories; Bayer AG
Customization Available Upon Request

Key Segments Profiled in the Diamond–Blackfan Anemia (DBAAASE) Syndrome Therapeutics Market Industry Survey 

By Treatment:

  • Corticosteroids
  • Chelation Therapy
  • Blood Transfusions
  • Stem Cell Transplant
  • Others

By End User:

  • Hospitals
  • Clinics
  • Others

By Region:

  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa

Frequently Asked Questions

How did the Market Develop in the Past?

The global market exhibited a 3.8% CAGR from 2018 to 2022.

What is the Future Growth Forecast for the Market?

The market is forecast to register a CAGR of 4.2% through 2033.

Which Treatment Segment is Expected to Witness High Demand?

Corticosteroids are among the most widely prescribed medicine classes worldwide.

What is another Treatment Segment Expected to Grow?

The blood transfusions segment is predicted to expand significantly.

What are the Challenges Projected to be Faced by the Market Players?

High costs and complicated side effects are likely to hinder market growth.

Table of Content
	1. Executive Summary | Diamond-Blackfan Anemia (DBA) Syndrome Therapeutics Market
	2. Market Overview
	3. Market Background
	4. Global Market Analysis 2018 to 2022 and Forecast, 2023 to 2033
	5. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Treatment
		5.1. Corticosteroids
		5.2. Chelation Therapy
		5.3. Blood Transfusions
		5.4. Stem Cell Transplant
		5.5. Others
	6. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By End User
		6.1. Hospitals
		6.2. Clinics
		6.3. Others
	7. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Region
		7.1. North America
		7.2. Latin America
		7.3. Europe
		7.4. Asia Pacific
		7.5. Middle East & Africa (MEA)
	8. North America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
	9. Latin America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
	10. Europe Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
	11. Asia Pacific Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
	12. MEA Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country
	13. Key Countries Market Analysis
	14. Market Structure Analysis
	15. Competition Analysis
		15.1. Sumitomo Corporation
		15.2. Johnson & Johnson Private Limited
		15.3. Novartis AG
		15.4. Pfizer Inc.
		15.5. Sanofi
		15.6. Merck KGaA
		15.7. LEO Pharma A/S
		15.8. Cipla Inc.
		15.9. GlaxoSmithKline plc
		15.10. AstraZeneca
		15.11. Abbott
		15.12. Bayer AG
	16. Assumptions & Acronyms Used
	17. Research Methodology
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