Thalassemia Market Size, Share & Trends Analysis Report By Therapeutic Class (Iron Chelating Drugs), By Pipeline (Gene Therapy), By Region (U.S., U.K., France, Germany, Italy, Spain, Japan), And Segment Forecasts, 2016 - 2022

Report Overview

The global thalassemia market size was valued at USD 1.72 billion in 2016 and is estimated to expand at a CAGR of 10.8% during the forecast period. Large population, increase in awareness about available treatment options, and rise in demand for improved curative gene therapies are some of the major factors expected to drive the market. 

Thalassemia is a rare hereditary blood disorder characterized by the production of abnormal hemoglobin, which is responsible for the destruction of red blood cells, leading to anemia. Blood transfusion and iron chelation therapies are the only treatment options available at present. This gives rise to ample opportunities in the market for the development of a universal curative treatment for thalassemia.

According to the Thalassemia International Federation, approximately 7.0% of the global population suffers from diseases and conditions associated with hemoglobin, out of which around 70.0% have sickle cell anemia and the remaining develop thalassemia or other blood disorders. Around 2.1% of the global population is living with various forms of thalassemia. This disorder affects approximately 4.4 in every 10,000 live births worldwide.

The disease is most prevalent in Greece, coastal regions of Turkey, some parts of Italy, major Mediterranean islands such as Cyprus, and South Asian countries such as Maldives and India. In the U.S., thalassemia is considered as an orphan or rare disease, which affects less than 20,000 people.

Therapeutic Class Insights

Currently, management of thalassemia involves symptomatic treatment rather than cure. Medications for the disease mainly address iron overload due to blood transfusions, vitamin supplements, fetal hemoglobin elevating drugs, and indicaxanthin-an antioxidant for the prevention of hemoglobin breakdown.

The market for thalassemia therapeutics is expected to exhibit strong commercial opportunity if drugs currently under phase II/III clinical pipeline receive regulatory approval. Increase in awareness about available treatment modalities and the introduction of novel treatment methods drives the market in developed regions such as North America and Europe. In addition, large youth population in emerging markets such as India and Pakistan is anticipated to boost demand for thalassemia drugs and treatments in the coming years.

Pipeline Insights

Gene therapy is expected to be immensely promising among other emerging curative therapies, with several genomic products in late-stage trials. Currently, there are 15 pipeline products under clinical trials for the treatment of thalassemia. Majority of these products are based on gene therapy and focus on providing a cure for the condition.

The report on the thalassemia market highlights promising pipeline drugs that are expected to be launched in the forecast period. Clinical trial results of these drugs reported encouraging top-line primary and secondary endpoint data. Gene therapy has the potential to change the thalassemia treatment landscape amid significant clinical development challenges and regulatory uncertainties.

Regional Insights

The EU5 countries held the largest share among the seven major markets, with a 38.0% share in 2016, followed by U.S. and Japan. Introduction of gene therapy for the treatment of thalassemia is expected to boost the market share of the U.S. and EU. In the rest of the world, the adoption of gene therapy for thalassemia treatment will be slower; however, significant growth is anticipated in the forecast period.

Beta-thalassemia is a common autosomal recessive disorder. It is relatively rare in the U.S. Approximately one in every 100,000 individuals in the global population shows symptoms of this disease. The disease is particularity prevalent in the Mediterranean region, Africa, Middle East, Central Asia, the Indian subcontinent, and the Far East.

Thalassemia Market Share Insights

Companies such as bluebird bio, Inc.; Acceleron Pharma, Inc.; Celgene Corporation; GlaxoSmithKline plc; Incyte Corporation; Gamida Cell; Kiadis Pharma; La Jolla Pharmaceutical Company; and IONIS Pharmaceuticals have several drugs in the pipeline, aiming to make treatments more accessible and affordable. 

Several novel gene therapies with enhanced mechanism of action are currently in late-stage trials and are aimed at addressing the strong unmet need for thalassemia treatment.

Thalassemia Market Report Scope

Segments Covered in the Report

This report forecasts revenue growth at country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2016 to 2022. For the purpose of this study, Grand View Research has segmented the global thalassemia market report on the basis of therapeutic class, pipeline, and region:

• Therapeutic Class Outlook (Revenue, USD Million, 2016 - 2022)

  • Iron Chelating Drugs

    • Exjade/Jadenu (deferasirox)

• Pipeline Outlook (Revenue, USD Million, 2016 - 2022)

  • Gene Therapy

    • LentiGlobin

• Regional Outlook (Revenue, USD Million, 2016 - 2022)

  • The U.S.

  • The U.K.

  • France

  • Germany

  • Italy

  • Spain

  • Japan