- In January 2024, Agios Pharmaceuticals announced that the global Phase 3 ENERGIZE study of mitapivat in patients with non-transfusion-dependent (NTD) alpha- or beta-thalassemia successfully met its primary endpoint of hemoglobin response. In addition, both critical secondary endpoints, including changes from baseline in the FACIT-Fatigue Score and hemoglobin concentration, were found to be statistically significant, underscoring the therapeutic potential of mitapivat for NTD thalassemia patients
- In January 2024, the U.S. FDA granted approval for Casgevy, a CRISPR-based gene therapy, for its second application in treating transfusion-dependent beta-thalassemia patients aged 12 and older. This follows Casgevy's December 2023 approval for use in treating sickle cell disease. The expanded approval marks a significant milestone in advancing gene-editing therapies for blood disorders, offering a promising alternative to regular transfusions and their associated complications
- In December 2023, Editas Medicine presented new safety and efficacy data from 17 patients treated with its CRISPR-based therapy, EDIT-301 (renizgamglogene autogedtemcel), as part of the EdiTHAL trial for transfusion-dependent beta thalassemia (TDT). The trial, with six patients, highlighted positive results, demonstrating the potential of EDIT-301 to offer a transformative treatment for TDT. The findings are significant in advancing gene-editing therapies for rare blood disorders
- In November 2023, Vertex Pharmaceuticals and CRISPR Therapeutics revealed that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) granted conditional marketing authorization for Casgevy (exagamglogene autotemcel, exa-cel), a CRISPR/Cas9 gene-edited therapy. This authorization, for the treatment of transfusion-dependent beta-thalassemia (TDT), highlights the growing role of gene-editing technologies in treating inherited blood disorders and offers patients an innovative, potentially curative option for managing TDT
- In August 2022, the FDA approved Zynteglo (beti-cel), the first curative gene therapy for patients with beta-thalassemia requiring regular red blood cell transfusions. Zynteglo represents a significant advancement in the treatment of beta-thalassemia, offering a one-time gene therapy that could potentially eliminate the need for lifelong transfusions. This approval marks a transformative moment in gene therapy, providing hope for patients suffering from this debilitating genetic disorder
- In February 2022, the FDA approved PYRUKYND (mitapivat) for treating adults with pyruvate kinase (PK) deficiency, a rare and lifelong hemolytic anemia. This approval marks a significant step in addressing PK deficiency, a condition that severely impacts red blood cell production. PYRUKYND provides a novel treatment option for patients in India and globally, helping improve quality of life by reducing the severity of anemia and its associated symptoms
- In April 2021, Vertex Pharmaceuticals and CRISPR Therapeutics announced a collaboration to develop, manufacture, and commercialize CTX001, a CRISPR/Cas9-based genome-editing therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). The agreement included a USD 900 million initial payment to CRISPR, with an additional USD 200 million contingent upon the first regulatory approval of CTX001. This partnership aims to bring transformative therapies to market for both SCD and TDT, offering hope for gene-based cures
Frequently Asked Questions
The market is segmented based on Segmentation, By Type (Alpha-thalassemia and Beta Thalassemia), Treatment (Blood Transfusions, Iron Chelation Therapy, Folic Acid Supplements, Gene Therapy, and Bone Marrow Transplants), End User (Hospitals and Clinics, Diagnostic Laboratories and Others) – Industry Trends and Forecast to 2032
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The Global Thalassemia Market size was valued at USD 986.00 USD Million in 2024.
The Global Thalassemia Market is projected to grow at a CAGR of 16% during the forecast period of 2025 to 2032.
The market report covers data from the U.S., Canada and Mexico in North America, Germany, France, U.K., Netherlands, Switzerland, Belgium, Russia, Italy, Spain, Turkey, Rest of Europe in Europe, China, Japan, India, South Korea, Singapore, Malaysia, Australia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific (APAC) in the Asia-Pacific (APAC), Saudi Arabia, U.A.E, South Africa, Egypt, Israel, Rest of Middle East and Africa (MEA) as a part of Middle East and Africa (MEA), Brazil, Argentina and Rest of South America as part of South America.
