Research Reports

Market Overview

• The global hemoglobinopathies market is expected to grow at a CAGR of over 10% during the forecast period (2019-2026).
• Hemoglobinopathy is a genetic disorder that causes abnormal production or aberration in the hemoglobin structure. Hemoglobinopathies are the most common type of Sickle Cell disease and are hereditary. The formation of the abnormal hemoglobin is observed due to structural defects in the hemoglobin molecule, diminished production of one of the two subunits of the hemoglobin molecule and abnormal associations of otherwise normal subunits. Hemoglobinopathies are formed due to defects in genes and anemia. Critical cases of hemoglobinopathy lead to dysfunction of the organ or death if it is left untreated. The disease has no permanent cure; this has led to increased demand in the pharmaceutical sector for new product innovations. The disorders can be severe or mild.
• According to WHO, hemoglobin disorders were originally endemic in 60% of 229 countries, potentially affecting 75% of births, but are now sufficiently common in 71% of countries among 89% of births. Around 1.1% of couples worldwide are at risk for having children with a hemoglobin disorder, and 2.7 per 1000 conceptions are affected.

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Market Dynamics

• The market is driven by rising cases of hemoglobin disorders such as sickle cell disease, thalassemia, Hb C, and Hb E, increase in R&D activities to develop new therapeutics, increase in awareness program by government and robust pipeline.
• Governments are collaborating with local institutes to start awareness programs for curbing the effect of hemoglobinopathies and related deaths. For instance, in 2016, The American Society of Hematology (ASH) founded Sickle Cell Disease Coalition (SCDC) to promote awareness for the condition and enhance outcomes for the individual. Similarly, in 2013, blood disorders division of the Centre for Disease Control and Prevention (CDC) along with the Association of Public Health Laboratories partnered on the hemoglobinopathies project for newborn screening health program. The project was launched to provide technical assistance to laboratories for assessment of hemoglobinopathies related complications and also imparting education to caregivers, healthcare workers, and patients on the hemoglobinopathy screening programs.
• The National Heart, Lung, and Blood Institute (NHLBI) which is part of National Institutes of Health (NIH) funds research programs for sickle cell diseases to develop new ways to detect and treat the condition and also improve patient outcomes. Advancements in gene-editing technologies are helping the development of new procedures, which involves editing or replacing the faulty gene and transplanting the corrected gene back to the patients. There is an increase in grants for prevention and treatment of sickle cell disease through the enactment of laws such as the Sickle Cell Disease Research, Surveillance, Prevention, and Treatment Act, of 2018 that is likely to help in better management of the condition.
• Insufficient healthcare infrastructure, no permanent cure, and lack of skilled professionals to interpret the complex data are going to restrain the market in the forecast period.

Pipeline

• Permanent cure for hemoglobinopathies is the biggest requirement for the treatment. The pipeline of drugs is promising and is expected to grow in the period of forecast.
• In December 2018, bluebird bio, Inc. announced new data from the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) clinical studies of its investigational LentiGlobin™ gene therapy in the treatment of patients with transfusion-dependent β-thalassemia (TDT) at the 60th Annual Meeting of the American Society of Hematology (ASH).
• In December 2018, Gamida Cell Ltd. announced the publication of data from the previously reported, multi-center Phase 1/2 clinical study evaluating the safety and efficacy of NiCord® as a stand-alone, hematopoietic stem cell (bone marrow) transplant in the Journal of Clinical Oncology. NiCord is an investigational product candidate in Phase 3 development as a universal bone marrow transplant solution for patients with high-risk hematologic malignancies, or blood cancers.

Market Segmentation

• The global hemoglobinopathies market can be segmented by type as Thalassemia, Sickle Cell Disease, and Hb Variants Diseases, by diagnoses test type as Genetic Testing, Isoelectric Focusing, High-Performance Liquid Chromatography, Hemoglobin Electrophoresis, and Molecular Methods, Molecular methods are further segmented as Polymerase Chain Reaction (PCR), Restriction Fragment Length Polymorphism (RFLP) and DNA Sequencing and by therapy type as Blood Transfusion, Bone Marrow Transplant, Iron Chelation Therapy and Folic Acid Supplements.
• By diagnosis test type Hemoglobin Electrophoresis is expected to grow at the highest pace over the period of the forecast as people who have thalassemia have fewer healthy red blood cells and less hemoglobin than normal in their blood, Hemoglobin tests measure the types of hemoglobin in a blood sample.
• By therapy, Blood Transfusion is expected to account for the largest segment over the period of forecast, owing to the high acceptance of this treatment option and significant success rate. Blood Transfusion of red blood cells are the main treatment for people who have moderate or severe thalassemia. This treatment gives healthy red blood cells with normal hemoglobin. The blood is given through a tube inserted into a vein in the arm. It is usually done in a hospital and takes a few hours each time. The frequency of blood transfusion depends on the type of thalassemia. Blood transfusions are very safe, but they can cause too much iron to build up in the body, so it requires medicine to remove the excess iron.

Geographical Analysis

• Geographically, the global hemoglobinopathies market is divided into North America, Europe, South America, Asia-Pacific, and the Middle East and Africa.
• North America is expected to account for the largest market share over the period of forecast due to the growing prevalence of blood disorders, favourable government programs, and high level of awareness among healthcare professionals and patients for hemoglobinopathies-related genetic testing.
• Asia Pacific is expected to grow at the highest CAGR over the period of the forecast as the prevalence of hemoglobinopathies is high in low-income countries of this region, such as According to United Nations (UN) data, 85% of the affected population in U.S. and Europe has an ancestral base in Asia and Africa regions. Asia has a large target population and rising economic levels. Moreover, the introduction of low-cost diagnostic alternatives for hemoglobinopathies is likely to further propel the market growth

Competitive Analysis

• Some of the major key players in the market are Gamida Cell, Alnylam Pharmaceuticals, Sanofi, Sangamo Therapeutics Inc., Global Blood Therapeutics, bluebird bio Inc., Emmaus Life Sciences Inc., Prolong Pharmaceuticals, Celgene Corporation, ApoPharma, Bristol-Myers Squibb, Emmaus Medical, Medunik USA, and Novartis.
• In February 2019, Gamida Cell Ltd. announced that translational data from the completed Phase 1/2 clinical study of NiCord® in an oral presentation at the 2019 Transplantation & Cellular Therapy (TCT) Meetings of American Society for Blood and Marrow Transplantation (ASBMT) and Center for International Blood and Marrow Transplant Research (CIBMTR). The data demonstrated that treatment with NiCord, an investigational advanced cell therapy designed to enhance and expand the life-saving benefits of bone marrow transplant for patients with hematologic malignancies, resulted in rapid and robust immune reconstitution.
• In January 2019, Gamida Cell Ltd. and Be The Match BioTherapies announced a strategic collaboration to improve outcomes for patients undergoing allogeneic hematopoietic stem cell (bone marrow) transplantation.
• In January 2019, Global Blood Therapeutics, Inc. announced successful data from its Phase 1/2 study of voxelotor in patients with sickle cell disease.
• In July 2018, Gamida Cell Ltd. announced that the U.S. Food and Drug Administration (FDA) had granted orphan drug designation for NiCord as a treatment for hematopoietic stem cell transplantation (HSCT).

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