Hemoglobinopathies Market Size, Share & Trends Analysis Report By Type (Thalassemia, Sickle Cell Disease, Other Hemoglobin (Hb) Variants), By Diagnosis, By Therapy, By Region- Global Industry Analysis, Share, Growth, Regional Outlook and Forecasts, 2023-2032

According to Nova one advisor, the global Hemoglobinopathies Market Size was valued at USD 10.1 billion in 2022 and is predicted to be worth USD 25.7 billion by 2032, with a CAGR of 13.80% from 2023 to 2032. 

Key Takeaways:

• Based on type, sickle cell disease accounted for the largest share of 58.6% in 2022 and is anticipated to maintain its lead over the forecast period
• The newborn patient population with sickle cell anemia is anticipated to reach 400,000 by 2050. Half of the global sickle cell anemia burden is present in India, Nigeria, and the Democratic Republic of the Congo
• By therapy, in the thalassemia segment, the blood transfusion segment held the largest market share in 2022 and is estimated to dominate the market throughout the forecast period
• Asia Pacific is expected to witness the fastest growth over the forecast period owing to a large potential patient base and high unmet medical needs in the region
• North America held the largest market share in 2022 due to government initiatives focusing on the development of new therapies and better reimbursement scenario

Increasing awareness regarding hemoglobinopathies and government initiatives to diagnose the diseases at an early stage are expected to propel the market growth over the forecast period. Moreover, increasing R&D investment, the presence of a promising drug pipeline, and technologically advanced diagnostics platforms are expected to boost the growth of the market.

The development of novel curative technologies, such as CRISPR/Cas9 and hematopoietic stem cell transplantation, coupled with a promising pipeline, is expected to propel market growth. Moreover, the presence of regulatory agencies, such as the FDA, which are working toward improving drug approval rate by granting accelerated approval for hemoglobinopathies drugs, is expected to drive the market. For instance, in 2019, the FDA has approved four novel drugs: Reblozyl by Bristol-Myers Squibb Company, Adakveo by Novartis AG, Zynteglo by bluebird bio, Inc., and Oxbryta by Global Blood Therapeutics, Inc. for the treatment of hemoglobinopathies.

Initiatives such as the Sickle Cell Awareness Initiative (SCAI) are working toward educating the people about the disease, which will increase the diagnosis and treatment rate. SCAI also raises funds for individuals affected with sickle cell diseases (SCDs) and provides research funding. Moreover, governments of Middle Eastern and Asian countries provide funds for R&D of hemoglobinopathies treatment. For instance, various programs undertaken by governments, such as thalassemia screening in neonates and providing medicines, are impacting the market growth positively.

The high patient population in low-income countries has encouraged market players and non-profit organizations to launch several initiatives to improve the access to the therapy. For instance, in February 2019, the Access to Excellent Care for Sickle Cell Patients Pilot Program (ACCEL) was launched by Global Blood Therapeutics, Inc. in order to provide research funding for novel SCD projects, with an aim to expand the access to optimal healthcare for sickle cell disease.

Biopharmaceutical companies are collaborating with nonprofit organizations to promote public awareness about hemoglobinopathies. For instance, in June 2018, the Sickle Cell Disease Association of America partnered with Emmaus Life Sciences, Inc. to provide online educational tools to sickle cell patients, friends, clinicians, caregivers, and researchers for spreading awareness about complications associated with SCD.

An increase in investment and funding for the development of novel therapies to treat hemoglobinopathies will further boost the market growth over the forecast period. The National Heart, Lung, and Blood Institute (NHLBI) has significantly invested in research & development in sickle cell disease. The NHLBI supports research work through various initiatives. Furthermore, private funding such as the Bronx Blood Research Fund (BBRF) provides a platform for research and management of thalassemia and other hemoglobinopathies.

Gene therapy has emerged as a promising treatment option for managing hemoglobin disorder as it targets the underlying genetic cause of the condition through the administration of one-time gene therapy and significantly reduces the need for blood transfusions. The current pipeline of gene therapy products includes CTX001 (CRISPR Therapeutics), BIVV003 (Sangamo Therapeutics, Inc. & Bioverativ Inc), and HGB-206 (bluebird bio, Inc).

Report Scope of the Hemoglobinopathies Market

Type Insights

The sickle cell disease segment held the largest share of 58.6% in 2022 and is expected to grow at the fastest rate during the forecast period. This is attributed to growing initiatives by biopharmaceutical companies and nonprofit organizations focused on improving access to SCD treatment. Furthermore, sickle cell disease affects around 300,000 newborns annually. An estimated 10% to 40% of the individuals in many African countries are carriers of sickle cell gene, which has resulted in a prevalence of around 2% in these countries. The increasing global disease burden is expected to fuel market growth over the forecast period.

The thalassemia segment is further sub-segmented into alpha-thalassemia and beta-thalassemia. Globally, 5% of the total population are carriers of alpha-thalassemia and around 1.5% of the population have beta-thalassemia traits. India has the maximum number of thalassemia carriers in the world. Moreover, biopharmaceutical companies are actively collaborating with each other to develop novel therapies for treating the disorder. For instance, in December 2017, CRISPR Therapeutics AG and Vertex Pharmaceuticals Incorporated entered into a partnership to develop and commercialize novel gene-editing treatments for thalassemia using CRISPR/Cas9 technology.

Diagnosis Insights

The sickle cell disease diagnosis segment dominated the market and accounted for more than 52.0% share in 2022 owing to high diagnostic rates in North America and Europe. Blood tests are most widely used for the diagnosis of SCD in patients. Moreover, the introduction of newborn baby screening programs for sickle cell disease in countries, such as the U.S., France, and Italy, will drive the segment at the fastest rate. In 2022, a call of action for the newborn screening was signed by many members of the European Parliament and health stakeholders for rare diseases.

The thalassemia diagnosis segment is estimated to grow at a significant rate over the forecast period. The introduction of technologically advanced, rapid, and user-friendly diagnostic tests is driving the segment. In addition, the presence of companies such as Oasis Diagnostics and LabCorp, which provide molecular diagnostic tests based on Multiplex Ligation-dependent Probe Amplification (MLPA) and Polymerase Chain Reaction (PCR) for screening and detection of alpha- and beta-thalassemia, is expected to fuel the segment growth.

Therapy Insights

The sickle cell disease segment held the largest revenue share of 61.4% in 2022. Extensive research has been carried out to develop novel therapies for treating the disorder, which is contributing to the segment growth. Gene therapy has emerged as a promising treatment option for managing the disorder as it targets the underlying genetic cause of the condition through one-time administration and reduces the need for patients to undertake blood transfusions. Robust product pipeline of gene therapy products, including CTX001 (CRISPR Therapeutics), BIVV003 (Sangamo Therapeutics, Inc. & Bioverativ Inc), and HGB-206 (bluebird bio, Inc), is expected to support segment growth.

Blood transfusion is considered to be the first line of treatment for hemoglobin disorders. The frequency of blood transfusion is higher in thalassemia cases as compared to other hemoglobinopathies. The transfusion is done every 3 to 4 weeks to help maintain the normal level of blood components. However, blood transfusion at frequent intervals increases the risk of acquiring infectious diseases and high blood iron levels. In 2022, the blood supply was interrupted because of a complete lockdown due to COVID-19 in many countries. The overall share of blood transfusion therapy and iron chelation therapy was estimated to decline due to the pandemic in the year 2022.

Bone marrow transplant (BMT) therapy is expected to expand at a significant growth rate over the forecast period. BMT is often used when blood transfusion and other therapies fail. BMT is reported to be effective when performed in the early stages of disease progression. It helps to provide healthy bone marrow to patients who are unable to produce a sufficient number of normal cells. BMT infuses healthy stem cells in the patient’s body to replace the diseased or damaged bone marrow and is increasingly being used to treat severe cases of sickle cell disease and thalassemia.

Regional Insights

North America held the largest revenue share of 40.2% in 2022 owing to increasing awareness of hemoglobinopathies among people and improving healthcare facilities. Various organizations spread awareness by conducting different programs. For instance, the Sickle Cell Disease Coalition creates awareness, funds research programs, and provides access to treatment in the U.S. Its membership includes research, public health, and provider organizations, faith-based organizations, industry representatives, foundations, patient groups, and federal agencies. Various initiatives undertaken by research organizations and government bodies to promote research to develop novel therapies for treating hemoglobinopathies are likely to contribute to the market growth in the region.

Moreover, increasing immigration to North America and Europe from high prevalence areas and improving diagnosis and treatment for common and rare genetic diseases, such as thalassemia and sickle cell disease, are some of the factors expected to fuel the market growth in these regions. The prevalence of hemoglobinopathies among migrated people in North America and Europe is high as compared to native people.

Asia Pacific is estimated to exhibit a lucrative growth rate over the forecast period. The presence of a large number of patients suffering from sickle cell disease and thalassemia is expected to propel market growth in the coming years. Favorable government initiatives, which are aimed at improving the standard of care provided to patients affected with hemoglobinopathies, are contributing to the market growth in the region. Due to improved healthcare facilities, the burden of SCD and thalassemia is growing in the region.

Some of the prominent players in the Hemoglobinopathies Market include:

• Sangamo Therapeutics, Inc.
• Global Blood Therapeutics, Inc.
• bluebird bio, Inc.
• Emmaus Life Sciences Inc.
• Pfizer, Inc.
• Novartis AG
• Prolong Pharmaceuticals, LLC
• Bioverativ Inc.
• Gamida Cell

Segments Covered in the Report

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2018 to 2032. For this study, Nova one advisor, Inc. has segmented the global Hemoglobinopathies market.

• Type

• Thalassemia
• Sickle Cell Disease
• Other Hemoglobin (Hb) Variants
• Diagnosis
• Thalassemia

• Blood Test
• Genetic Test
• Prenatal Genetic Test
• Pre-implantation Genetic Diagnosis
• Electrophoresis
• Others
• Sickle Cell Disease
• Blood Test
• Genetic Test
• Prenatal Genetic Test
• Electrophoresis
• Others
• Other Hemoglobin (Hb) Variants
• Blood Test
• Genetic Test
• Prenatal Genetic Test
• Electrophoresis
• Others
• Therapy
• Thalassemia
• Blood Transfusion
• Iron Chelation Therapy
• Bone Marrow Transplant
• Others
• Sickle Cell Disease
• Blood Transfusion
• Hydroxyurea
• Bone Marrow Transplant
• Others
• Other Hemoglobin (Hb) Variants
• Blood Transfusion
• Hydroxyurea
• Iron Chelation Therapy
• Bone Marrow Transplant
• Others

By Geography

• North America
• Europe
• Asia-Pacific
• Latin America
• Middle East & Africa (MEA)

Key Benefits for Stakeholders

• This report provides a quantitative analysis of the market segments, current trends, estimations, and dynamics of the global Hemoglobinopathies industry analysis from 2023 to 2032 to identify the prevailing Hemoglobinopathies industry opportunity.
• The market research is offered along with information related to key drivers, restraints, and opportunities.
• Porter's five forces analysis highlights the potency of buyers and suppliers to enable stakeholders make profit-oriented business decisions and strengthen their supplier-buyer network.
• In-depth analysis of the global Hemoglobinopathies industry segmentation assists to determine the prevailing market opportunities.
• Major countries in each region are mapped according to their revenue contribution to the global market.
• Market player positioning facilitates benchmarking and provides a clear understanding of the present position of the market players.
• The report includes the analysis of the regional as well as global Hemoglobinopathies industry trends, key players, market segments, application areas, and market growth strategies.