Beta Thalassemia – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)

Beta thalassemia (BT) is a rare genetic disorder characterized by the reduced production of hemoglobin. BT minor is caused by a mutation in one hemoglobin beta (HBB) gene, and transfusion-dependent (TD) thalassemia is caused by a mutation in both HBB genes, although some patients with mutations in both genes are classified as having non-transfusion-dependent (NTD) thalassemia. Patients can present with jaundice, bone deformities, and chronic anemia. Management of BT was once limited to regular blood transfusions and iron chelators, but therapies such as BMS’s Reblozyl, Bluebird Bio’s Zynteglo, and Vertex / CRISPR Therapeutics’ Casgevy now offer a more promising future for BT patients. Nevertheless, strong unmet need remains for universally curative therapies that improve BT patients’ quality of life. In this report, we examine the current treatment of BT in the United States and EU5 countries and the agents in development, including those that target the underlying genetic defect and reduce the transfusion burden.

Questions answered

• How large is the diagnosed BT population in the United States and EU5? How will the population change through 2034?
• What is the current landscape for BT patients, and how will it change over the next 10 years considering alternative therapies, such as Zynteglo and Casgevy, are now available?
• What pipeline products are most promising, and what sales / uptake could they secure in BT? How will emerging therapies affect medical practice?

Product description

Niche & Rare Disease Landscape & Forecast offers comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research, enabling you to:

• Optimize your long-term disease and development strategy.
• Quantify market potential for your pipeline assets and those of your competitors.
• Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.
• Gauge the commercial outlook and impact of key market events.

Content highlights

Publication date September 2025

Geography United States, EU5

Primary research

Country-specific interviews with thought-leading hematologists

Survey data collected for this and other Clarivate research

Epidemiology

Diagnosed prevalent and drug-treatable cases of beta thalassemia by country, segmented by severity, genotype, and availability of HLA-matched sibling HSCT donor

Forecast

Drug-level sales and patient share of key beta thalassemia therapies in 2024 and 2034

Drug treatments

Coverage of key current and late-phase emerging therapies