Hemoglobinopathy includes a range of disorders, which occur due to altered structure of hemoglobin. Hemoglobin is present in the red blood cells that transport oxygen from the lungs to the body. Hemoglobinopathy disorder is inherited and are passed on from one generation to another. It is further classified into two categories – thalassemia syndrome and structural hemoglobin variants. Thalassemia syndrome includes disorder related to synthesis defect. The synthesis defect of alpha globin gives rise to alpha thalassemia and defect in beta globin give rise to beta thalassemia. The structural hemoglobin variants occur due to alteration of alpha and beta chains that cause defect in structure.
Hemoglobinopathy Treatment Drugs Market - Impact of Coronavirus (Covid-19) Pandemic
The pandemic of COVID-19 has significantly augmented demand for several drugs. The correct treatment and management of infection by coronavirus (COVID-19) in patients affected by rare hematological diseases may be challenging due to the rapid spread of the pandemic and limited literature so far, especially in some countries such as U.K, U.S. Italy, Face, Spain, Russia, India and others. Patients with certain hemoglobinopathies are more prone to get infected by coronavirus infection. For instance, in June 2020, according to survey carried out by European Hematology Association (EHA) Annual Congress, many patients with hemoglobinopathies, predominantly those with sickle cell disease, were infected with severe acute respiratory syndrome coronavirus.
The hemoglobinopathy treatment drugs market is estimated to be valued at US$ 3.3 billion in 2020 and is expected to exhibit a CAGR of 8.4% during the forecast period (2020-2027).
Figure 1. Hemoglobinopathy Treatment Drugs Market Share (%) Analysis, By Drug Type, 2020
Increasing prevalence of hemoglobinopathies and product approvals are expected to boost growth of the hemoglobinopathy treatment drugs market
The rising prevalence of hemoglobinopathies such as thalassemia is expected to propel the hemoglobinopathy treatment drugs market growth over the forecast period. For instance, according to a report of Pediatric Blood Cancer 2018, alpha thalassemia is the most common single-gene disorder in the world. It is estimated that there are 270 million carriers of mutant globin genes that can potentially cause severe forms of thalassemia. Moreover, 300,000-400,000 severely affected infants are born every year, more than 95% of whom are in Asia, or in the Middle East.
Furthermore, the hemoglobinopathy treatment drugs market is expected to witness significant growth in the near future, owing to increasing product approvals by regulatory bodies. For instance, in November 2019, Celgene Corporation and Acceleron Pharma Inc. announced that the U.S. Food and Drug Administration (FDA) approved REBLOZYL (luspatercept-aamt) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. REBLOZYL is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia.
Hemoglobinopathy Treatment Drugs Market - Restraints
Failing clinical trials for the treatment of sickle cell anemia is expected to hinder the hemoglobinopathy treatment drugs market growth. For instance, in August 2019, Pfizer Inc. announced that the Phase III Rivipansel pivotal study, to evaluate the efficacy and safety of rivipansel in patients aged six and older with sickle cell disease, failed to achieve its primary or key secondary efficacy endpoints for pediatric patients.
Moreover, lack of a permanent cure for hemoglobinopathy disorders is also expected to limit the market growth. Most treatment options that are available focus on reducing symptoms of the disorders and the only reliable treatment available is bone marrow transplant, which is a very complex and expensive procedure and is also associated with some side effects.
|Base Year:||2019||Market Size in 2019:||US$ 3.3 Bn|
|Historical Data for:||2016 to 2019||Forecast Period:||2020 to 2027|
|Forecast Period 2020 to 2027 CAGR:||8.4%||2027 Value Projection:||US$ 6.4 Bn|
Bristol-Myers Squibb Company, GlycoMimetics Inc., Pfizer Inc., Anthera Pharmaceuticals Inc., Eli Lilly and Company, Mast Therapeutics, Daiichi Sankyo Company Ltd., Novartis International AG, Bluebird Bio Inc., HemaQuest Pharmaceuticals Inc., Emmaus Medical Inc., Prolong Pharmaceuticals, Merck & Co., Inc., Medunik USA Inc., Sangamo Therapeutics, Inc., Global Blood Therapeutics, Alnylam Pharmaceuticals, and Acceleron Pharma, Inc.
|Restraints & Challenges:||
Hemoglobinopathy Treatment Drugs Market – Regional Analysis
North America is estimated to account for the largest share in the hemoglobinopathy treatment drugs market in 2020, owing to increasing product approvals by regulatory bodies in North America region. For instance, in May 2020, CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
Furthermore, the increasing prevalence of hemoglobinopathies in Asia Pacific, Middle East, and Africa region is expected to drive the hemoglobinopathy treatment drugs market growth in the near future. For instance, in 2018, according to a report of Pediatric Blood Cancer 2018, the frequency of alpha thalassemia alleles in genes is 5-10% in the Mediterranean basin, 20-30% in portions of West Africa, and as high as 60-80% in parts of Saudi Arabia, India, Thailand, Papua New Guinea, and Melanesia. In Thailand, which has a population of 62 million people, around 7,000 infants are born each year with hemoglobin H disease.
Figure 2. Hemoglobinopathy Treatment Drugs Market Value (US$ Mn) & Y-o-Y Growth (%), 2017-2027
Hemoglobinopathy Treatment Drugs Market - Competitive Landscape
Key players operating in the hemoglobinopathy treatment drugs market include Bristol-Myers Squibb Company, GlycoMimetics Inc., Pfizer Inc., Anthera Pharmaceuticals Inc., Eli Lilly and Company, Mast Therapeutics, Daiichi Sankyo Company Ltd., Novartis International AG, Bluebird Bio Inc., HemaQuest Pharmaceuticals Inc., Emmaus Medical Inc., Prolong Pharmaceuticals, Merck & Co., Inc., Medunik USA Inc., Sangamo Therapeutics, Inc., Global Blood Therapeutics, Alnylam Pharmaceuticals, and Acceleron Pharma, Inc.
Hemoglobinopathy is a group of hereditary blood disorders involving an abnormality in the structure of hemoglobin. These disorders include both sickle cell disease (SCD) and thalassemia. Common symptoms of the condition include severe pain, shortness of breath, enlarged spleen, and growth problems in children. Other types of hemoglobinopathies linked to the structural hemoglobin variants are HbS, HbE, and HbC. The highly variable clinical manifestations of the hemoglobinopathies range from mild hypochromic anemia to moderate hematological disease to severe, lifelong, transfusion-dependent anemia with multiorgan involvement. Drugs to treat the symptoms of hemoglobinopathy include analgesics, antibiotics, ACE inhibitors, and hydroxyurea. According to the data of National Center for Biotechnology Information, more than 90% of patients currently survive the adulthood. Optimally treated patients have a projected life span of 50 to 60 years.
Increasing incidence and prevalence of sickle cell disease (SCD) is expected to propel growth of the global hemoglobinopathy treatment drugs market over the forecast period. For instance, according to the data published in Centers for Disease Control and Prevention in October 2019, SCD affects around 100,000 Americans every year. Moreover, as per the same source, around 1 in 13 African-American babies are born with sickle cell trait (SCT).
However, failing clinical trials for the treatment of sickle cell anemia are expected to hinder the market growth. For instance, in August 2019, Pfizer Inc. announced that the Phase III Rivipansel pivotal study, to evaluate the efficacy and safety of rivipansel in patients aged six and older with sickle cell disease, failed to achieve its primary or key secondary efficacy endpoints. Moreover, high costs, and difficulties associated with diagnosis of hemoglobinopathies such as lack of proper knowledge and lack of skilled professionals are expected to restrain growth of the market up to certain extent.
Key features of the study:
“*” marked represents similar segmentation in other categories in the respective section.