Hemoglobinopathies - Market Insights, Competitive Landscape, and Market Forecast - 2030

Hemoglobinopathies Market By Indication (Thalassemia, Sickle Cell Anaemia, And Others), By Treatment (Stem Cell Therapy, Blood Transfusion, Iron Chelation Therapy, And Others), and by geography is expected to advance at a respectable CAGR forecast till 2030 owing to the increasing prevalence of various hemoglobin disorders and the increasing research & developmental activities concerning to the hemoglobinopathies across the globe

The global hemoglobinopathies market will grow at a CAGR of 8.19% during the forecast period from 2024 to 2030 to reach USD 12.21 million by 2030. The hemoglobinopathies market is observing substantial market growth primarily due to the growing prevalence of various hemoglobin disorders such as sickle cell anemia, hemoglobin C disease, hemoglobin S-C disease, thalassemia, and others across the globe. Further, the presence of a robust number of Hemoglobinopathies in the pipeline, and the increasing focus of governments and healthcare organizations on rare diseases such as sickle cell anemia, thalassemia, and others will aid in surging the demand for Hemoglobinopathies in the upcoming years. Likewise, the increasing research & developmental activities along with the surging drug approvals and launches and the presence of key players in the market will create a requisite for hemoglobinopathies in the market. Therefore, the market for hemoglobinopathies is estimated to grow at a substantial CAGR during the forecast period from 2024 to 2030.

Hemoglobinopathies Market Dynamics:

According to the World Health Organization (WHO) 2022 estimates each year over 300,000 babies with severe forms of diseases are born worldwide, the majority in low and middle-income countries. Also, approximately 5% of the world's population are healthy carriers of a gene for sickle-cell disease (SCD) or thalassemia. The percentage of people who are carriers of the gene is as high as 25% in some regions. Further, these conditions are most prevalent in tropical regions; however, population migration has spread these diseases to most countries. As per the same source, Thalassaemia is one of the most common diseases in Asia, the Mediterranean basin, and the Middle East and Sickle-cell disease predominates in Africa.

Furthermore, the Centers for Disease Control and Prevention (CDC) in the latest data stated that sickle cell anemia affects approximately 100,000 Americans, every year. Also, SCD occurs among about 1 out of every 365 Black or African-American births and SCD occurs among about 1 out of every 16,300 Hispanic-American births.

Moreover, the clinical trials of hemoglobinopathies are expected to significantly advance the drug development process. For instance, in August 2022, Vertex Pharmaceuticals Incorporated started a Phase IIIb study to evaluate the efficacy and safety of a single dose of autologous CRISPR Cas9 modified CD34+ human hematopoietic stem and progenitor cells (CTX001) in subjects with transfusion-dependent B-thalassemia or severe sickle cell disease. The trial is anticipated to get completed by February 2025. Therefore, such trials will increase the chance of accessibility of hemoglobinopathies drugs during the forecast period which may create a rewarding chance for the key players to cater to the maximum market and make enormous revenue. Henceforth, the market of hemoglobinopathies will grow during the forecast period from 2022 - 2028.

Lastly, the rising investment in the development of novel therapies is further expected to provide impetus to market growth in the upcoming years. Also, major biopharmaceutical players are collaborating to develop novel therapeutics for hemoglobinopathies using the latest technology platforms. For instance, Sangamo Therapeutics, Inc. and Bioverativ have collaborated to develop therapeutics for hemoglobinopathies.

Therefore, the factors stated above collectively will drive the overall hemoglobinopathies market throughout the forecast period from 2024-2030.

However, the high cost of drug development and launches, low awareness regarding hemoglobinopathies, and stringent regulatory approval process may halt the market growth of hemoglobinopathies.

The hemoglobinopathies market was moderately impacted during the period of the COVID-19 pandemic. During the initial stage of the pandemic, the sale of hemoglobinopathies suffered collapse owing to the imposition of strict lockdown rules, disruption in manufacturing, and supply. However, the government classified medicines and drugs under emergency products during the pandemic, thereby leading to an increase in their demand or sales. However, the ease of lockdown restrictions, the upturn of resources and supply chain, the administration of a vaccine to the public, and with the increasing number of treatment rates for hemoglobin disorders, the demand for hemoglobinopathies increases and is anticipated to do the same during the forecast period from 2022 - 2028.

Hemoglobinopathies Market Segment Analysis:

Hemoglobinopathies Market by Indication (Thalassemia, Sickle Cell Anaemia, and Others), Treatment (Stem Cell Therapy, Blood Transfusion, Iron Chelation Therapy, and Others), and Geography (North America, Europe, Asia-Pacific, and Rest of the World).

In the treatment segment of the hemoglobinopathies market, the stem cell therapy segment is expected to have a significant revenue share in the year 2023. This was primarily owing to the efficiency offered by stem cell therapy in the treatment of hemoglobinopathies. Moreover, stem cell therapy also offers lesser side effects as compared to other treatments. Also, the expanding launches and strategic investments, and other activities by the key players will drive the market growth during the forecast period.

For instance, stem cell therapy is generally only considered in children with sickle cell disease who have severe symptoms that have not responded to other treatments, when the long-term benefits of a transplant are thought to outweigh the possible risks.

Also, stem cell therapy reduces disease burden, improves outcomes and quality of life for patients with SCD, and significantly reduces health care costs over the long term.

Moreover, the presence of a robust number of stem cell therapy treating hemoglobinopathies in the pipeline will in turn increase the market opportunity. For instance, in February 2020, bluebird bio started a Phase III study evaluating gene therapy by transplantation of autologous CD34+ stem cells transduced ex vivo with the BB305 lentiviral vector in subjects with sickle cell disease. The study is aimed to evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).

Therefore, owing to the above-mentioned factors, the demand for stem cell therapy upsurges, thereby the category is expected to witness considerable growth eventually contributing to the overall growth of the global hemoglobinopathies market during the forecast period.

Europe is expected to dominate the overall Hemoglobinopathies Market:

Among all the regions, Europe is expected to dominate global hemoglobinopathies in the year 2023 and is expected to do the same during the forecast period from 2024-2030.

This can be ascribed to the increasing demand for hemoglobinopathies in the region owing to the increasing prevalence of various hemoglobin disorders in the region. Further, the presence of key domicile players in the region, surging drug approvals and launches, and others are among the key factors that contribute to the growth of the hemoglobinopathies market in Europe during the forecast period from 2022 - 2028.

According to the 2020 report, Overview of the sickle cell disease environment in select European countries, Sickle cell disease (SCD) is a rare, inherited disorder, affecting more than 52,000 people in Europe, in 2020. It is the most prevalent genetic disease in France, and in the United Kingdom (UK), and its prevalence is increasing in many other European countries. Further, according to figures from the European Medicines Agency (EMA), in 2019 approximately 1 in 10,000 people lived with SCD in the EU/ European Economic Area (EEA).

Furthermore, the increasing activities by the key players and government in the region will in turn increase the demand for the product during the forecast period. For instance, in 2019, the European Joint Programme on Rare Diseases (EJP RD) was launched, to create an ecosystem for research, care, and innovation for rare diseases including hemoglobinopathies. The project has a total of Euro 100 million investment, Euro 55 million of which are contributed by the EU. It includes projects on translational research for rare diseases (translating pre-clinical research to clinical) among others.

Moreover, the market players in the country are continuously concentrating on the development of novel drugs to tackle mental disorders indications using hemoglobinopathies, and they are also adopting various strategies to develop better drugs with reduced side effects. Thus, this is likely to drive the market in the region. For instance, in October 2022, Global Blood Therapeutics started a randomized, double-blind, placebo-controlled, multicenter Phase III study to assess the safety and efficacy of Inclacumab in participants with sickle cell disease experiencing vaso-occlusive crises. The trial is being conducted in Germany, France, Italy, and various other European and North American Locations.

Therefore, the interplay of the aforementioned factors above would provide a conducive growth environment for the Europe region in the hemoglobinopathies market.

Hemoglobinopathies Market Key Players:

Some of the key market players operating in the hemoglobinopathies market are Global Blood Therapeutics, Inc., Sanofi, bluebird bio, Inc., Bristol-Myers Squibb Company, Emmaus Medical, Inc, Novartis, Merck KGaA, Chiesi USA, Inc., ApoPharma Inc., Alvogen., and others.

Recent Developmental Activities in the Hemoglobinopathies Market:

In August 2022, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

In November 2019, the U.S. Food and Drug Administration granted approval to Reblozyl (luspatercept-aamt) for the treatment of anemia (lack of red blood cells) in adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

In November 2019, the U.S. Food and Drug Administration (FDA) approved ADAKVEO (crizanlizumab-tmca) to reduce the frequency of vasoocclusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease.

In November 2019, the U.S. Food and Drug Administration granted accelerated approval to Oxbryta (voxelotor) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older.

Key Takeaways from the Hemoglobinopathies Market Report Study

Market size analysis for current hemoglobinopathies market size (2023), and market forecast for 5 years (2024-2030)

The effect of the COVID-19 pandemic on this market is significant. To capture and analyze suitable indicators, our experts are closely watching the hemoglobinopathies market.

Top key product/services/technology developments, mergers, acquisitions, partnerships, and joint ventures happened for the last 3 years

Key companies dominating the global hemoglobinopathies market.

Various opportunities available for the other competitor in the hemoglobinopathies market space.

What are the top-performing segments in 2023? How these segments will perform in 2030.

Which are the top-performing regions and countries in the current hemoglobinopathies market scenario?

Which are the regions and countries where companies should have concentrated on opportunities for hemoglobinopathies market growth in the coming future?

Target Audience who can be benefited from this Hemoglobinopathies Market Report Study

Hemoglobinopathies providers

Research organizations and consulting companies

Hemoglobinopathies -related organizations, associations, forums, and other alliances

Government and corporate offices

Start-up companies, venture capitalists, and private equity firms

Distributors and Traders dealing in hemoglobinopathies

Various end users who want to know more about the hemoglobinopathies market and the latest developments in the hemoglobinopathies market.

Frequently Asked Questions for the Hemoglobinopathies Market:

1. What are hemoglobinopathies?

Hemoglobinopathies are a group of disorders passed down through families (inherited) in which there is abnormal production or structure of the hemoglobin molecule.

2. What is the global market for hemoglobinopathies?

The global hemoglobinopathies market will grow at a CAGR of 8.19% during the forecast period from 2024 to 2030 to reach USD 12.21 million by 2030.

3. What are the drivers for the global Hemoglobinopathies market?

The hemoglobinopathies market is witnessing positive market growth owing to the factors such as the growing prevalence of hemoglobinopathies, the presence of a robust number of pipeline products across the globe, increasing focus of governments and health care organizations on rare diseases such as sickle cell anemia, thalassemia, and others, surging new drug launches and approvals, presence of key players in the market, and others will create an exigency for the Hemoglobinopathies market.

4. Who are the key players operating in the global hemoglobinopathies market?

Some of the key market players operating in the hemoglobinopathies market include Global Blood Therapeutics, Inc., Sanofi, bluebird bio, Inc., Bristol-Myers Squibb Company, Emmaus Medical, Inc, Novartis, Merck KGaA, Chiesi USA, Inc., ApoPharma Inc., Alvogen., and others.

5. Which region has the highest share of the hemoglobinopathies market?

Among all the regions, Europe is estimated to hold a significant revenue share in the global Hemoglobinopathies market. This can be ascribed to the increasing demand for hemoglobinopathies among the patients in the region owing to the increasing prevalence of various hemoglobin disorders such as sickle cell anemia, hemoglobin C disease, hemoglobin S-C disease, thalassemia, and others. Further, the presence of key domicile players and rising new drug approvals in the region are the reasons which act as a supportive factor for the Europe hemoglobinopathies market growth.