North America Hemoglobinopathies Market Report and Forecast 2024-2032

North America Hemoglobinopathies Market Outlook

North America hemoglobinopathies market size is expected to grow at a CAGR of 5.10% during the forecast period of 2024-2032, driven by the growing prevalence of sickle cell anemia and thalassemia along with the rising advancements in treatment and diagnostics in the region.

Key Takeaways

• In the United States, around 100,000 individuals are affected by sickle cell disease. It is highly prevalent in African Americans, affecting 1 out of every 365 African American births.
• In December 2023, the FDA approved the first cell-based gene therapies, Casgevy and Lyfgenia, to treat sickle cell disease (SCD) in patients aged 12 years and more.
• In July 2023, the National Institutes of Health awarded a USD 2.6 million grant to a Rice University research team to investigate the efficacy and safety of gene editing technology to treat sickle cell disease.

North America Hemoglobinopathies Market Analysis

Hemoglobinopathies are a group of inherited disorders characterized by the abnormal structure or production of the hemoglobin molecule. Sickle cell anemia and thalassemia are some common types of hemoglobinopathies. In the United States, around 100,000 individuals are affected by sickle cell disease. It is highly prevalent in African Americans, occurring in 1 out of every 365 African American births. The rising prevalence of sickle cell anemia disease is expected to increase demand for effective treatment options, driving North America hemoglobinopathies market growth.

One of the major market trends is the development of novel therapies to combat the growing cases of hemoglobinopathies. Furthermore, the rise in regulatory approvals by health regulatory bodies including United States Food and Drug Administration (FDA) to address the unmet of clinical needs of a large patient base is likely to boost market size in the region. In December 2023, the FDA granted approval to the first cell-based gene therapies, Casgevy and Lyfgenia, to treat sickle cell disease (SCD) in patients aged 12 years and more. Such innovations along with their high acceptance rate is projected to augment the North America hemoglobinopathies market share in the forecast period.

Casgevy, developed by Vertex Pharmaceuticals Incorporated, and a Swiss-American biotechnology CRISPR Therapeutics AG, is the first FDA-approved CRISPR/Cas9 therapy for the treatment of sickle cell disease in patients with recurrent vaso-occlusive crises. On the other hand, Bluebird Bio, Inc.’s Lyfgenia is a cell-based gene therapy that utilizes a lentiviral vector for gene delivery. It is used for the treatment of sickle cell disease in patients showing a history of vaso-occlusive events. The approval of these novel gene therapies which have the potential to cure the inherited disease is anticipated to fuel the North America hemoglobinopathies market demand.

The market is also driven by increased investments from both government and private sectors to support research activities aimed at the advancement of diagnostic methods and treatment of hemoglobinopathies. For instance, in July 2023, the National Institutes of Health awarded a USD 2.6 million grant to a Rice University bioengineer and his research team. The 4-year grant was intended to be used in investigating the efficacy and safety of gene editing technology to treat sickle cell disease. The rise in funding will help in the development of new treatment therapies which will propel the market growth in the region.

North America Hemoglobinopathies Market Segmentation

The report offers a detailed analysis of the market based on the following segments:

Market Breakup by Diagnosis

• Red Blood Cell (RBC) Count Test
• Genetic Testing
• High Performance Liquid Chromatography (HPLC) Test
• Hemoglobin Isoelectric (Hb IEF) Focusing
• Hemoglobin Electrophoresis (Hb ELP) Test
• Hemoglobin Solubility Test

Market Breakup by Type

• Thalassemia
• Sickle cell Disease
• Others

Market Breakup by Therapy

• Monoclonal Antibody Medication
• Ace Inhibitors
• Hydroxyurea
• Others

Market Breakup by Treatment

• Allogeneic Stem Cell Therapy
• Autologic Stem Cell Therapy
• Syngeneic Stem Cell Therapy

Market Breakup by Distribution Channel

• Hospital Pharmacy
• Online Providers
• Drug Store and Retail Pharmacy
• Others

Market Breakup by End User

• Biopharmaceutical & Biotechnology Companies
• Academic & Research Institutes
• Others

Market Breakup by Countries

• United States of America
• Canada

North America Hemoglobinopathies Market: Competitor Landscape

The key features of the market report include patent analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players. The major companies in the market are as follows:

• Sangamo Therapeutics, Inc.
• Global Blood Therapeutics
• Bluebird bio, Inc.
• Emmaus Life Science Inc.
• Pfizer, Inc.
• Novartis AG
• Prolong Pharmaceuticals, LLC
• Bioverativ Inc.
• Gamida Cell
• Celgene Corporation

Kindly note that this only represents a partial list of companies, and the complete list has been provided in the report.

FAQs

What is the North America hemoglobinopathies market forecast outlook for 2024-2032?
The market is anticipated to grow at a CAGR of 5.10% during the forecast period of 2024-2032, driven by the growing prevalence of sickle cell anemia and thalassemia in the region.

What are the major factors aiding the North America hemoglobinopathies market demand?
The rising advancements in the treatment and diagnostics of hemoglobinopathies are fuelling the demand for the market.

What are the major North America hemoglobinopathies market trends?
One of the significant trends in the market is the rise in regulatory approvals by the health regulatory body United States Food and Drug Administration (FDA). In December 2023, Casgevy and Lyfgenia, the first cell-based gene therapies received FDA approval for the treatment of sickle cell disease (SCD) in patients aged 12 years and more.

What is the market segmentation based on diagnosis?
Based on the diagnosis, the market is segmented into red blood cell (RBC) count test, genetic testing, high performance liquid chromatography (HPLC) test, hemoglobin isoelectric (Hb IEF) focusing, hemoglobin electrophoresis (Hb ELP) test, and hemoglobin solubility test.

What are the various treatments available in the market?
Treatments available in the market include allogeneic stem cell therapy, autologous stem cell therapy, and syngeneic stem cell therapy.

What is the market breakup by type?
By type, the market is divided into thalassemia, and sickle cell disease, among others.

What is the market segmentation based on therapy?
The market breakup by therapy includes monoclonal antibody medication, ace inhibitors, and hydroxyurea, among others.

What are the major end users of the market?
End users of the market are biopharmaceutical & biotechnology companies, academic & research institutes, among others.

What are the major distribution channels of the market?
Major distribution channels of the market include hospital pharmacies, online providers, drug stores, and retail pharmacies, among others.

What is the market segmentation by countries?
The market segmentation by countries includes the United States of America and Canada.

Who are the key players involved in the North America hemoglobinopathies market?
The key players in the market are Sangamo Therapeutics, Inc., Global Blood Therapeutics, Bluebird bio, Inc., Emmaus Life Science Inc., Pfizer, Inc., Novartis AG, Prolong Pharmaceuticals, LLC, Bioverativ Inc., Gamida Cell, and Celgene Corporation.


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