Beta Thalassemia | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2022

Beta thalassemia (BT) is a rare genetic disorder characterized by the reduced production of hemoglobin. It is caused by a mutation in one hemoglobin beta (HBB) gene (beta thalassemia minor) or a mutation in both HBB genes (beta thalassemia major; some patients with mutations in both genes can be classified as beta thalassemia intermedia). The mutation can cause jaundice, failure to thrive, and chronic anemia. Management of BT was once limited to regular blood transfusions and iron chelators; however, more-recent therapies such as Bristol Myers Squibb / Acceleron’s Reblozyl and Bluebird Bio’s Zynteglo offer a more-promising future. Reblozyl, an erythropoietin maturation therapy, launched in the United States in late 2019 and in Europe beginning in 2020 to treat anemia in adult patients. Zynteglo, a LentiGlobin vector-based gene therapy, is expected to launch in the United States in 2022; the therapy was available in Europe but was withdrawn by Bluebird Bio because of unfavorable pricing negotiations. Strong unmet need remains for universally curative therapies that improve patients’ quality of life. Drug developers are focusing on agents that target the underlying genetic defect and reduce the transfusion burden.

Questions Answered:

•How large is the diagnosed prevalent BT population in the United States and EU5? How will the population change through 2031?

•What is the current treatment landscape for BT patients, and how will it change in the next 10 years? How will the availability of Reblozyl and Zynteglo affect BT treatment?

•What pipeline products are most promising, and what sales/uptake could they secure in BT? How will new therapies affect medical practice?

GEOGRAPHIES

United States, EU5

PRIMARY RESEARCH

Six country-specific interviews with thought-leading hematologists

Supported by survey data collected for this study

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 shares of key beta thalassemia therapies through 2031, including Reblozyl and Zynteglo

EMERGING THERAPIES

Phase II: 6 drugs; coverage of select early-phase products

Marketed drugs: 2

Product Description

Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.