Thalassemia - A Pipeline Analysis Report

Overview of the drug development pipeline for Thalassemia

Thalassemia is an inherited disorder that can be inherited from one of the parents or both the parents. The two types of Thalassemia include alphas-thalassemia and beta-thalassemia. Patients suffering from this condition experience anemia, which is caused by excessive destruction of red blood cells. Fatigue, abdominal swelling, dark urine, pale skin, weakness are some of the symptoms that are experienced by patients suffering from Thalassemia. Technavio’s market research analysts have predicted that with the introduction of regular blood transfusion, gene transference, and cell replacement as some of the most promising approaches to treat Thalassemia, the global Thalassemia market will witness growth in the forthcoming years.

According to this pipeline analysis report, most of the drug development molecules in the pipeline are under the pre-clinical developmental stage. For instance, sponsors such as Aevi Genomic Medicine developed TARGTEPO and Agios developed AG-348, which are under pre-clinical development stage. The pipeline landscape in the pipeline analysis report mentions the percentage of drug development molecules that are under phase I, phase I/II, and phase II stages. Our market research analysts have also identified that a small amount of drug molecules that are being developed comes under phase III and discovery developmental stages.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are involved in the development of drug molecules for the treatment of Thalassemia. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug development molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are –

• Celgene
• Invenux
• Rare Partners

Therapeutic assessment of the drug development pipeline for Thalassemia by route of administration

• Oral
• Subcutaneous
• Intravenous
• Intraosseous

The oral route of administration (ROA) involves the administration of drug substances through the mouth cavity. It has been observed that the majority of total therapeutics are being developed for oral administration.

Therapeutic assessment of the drug development pipeline for Thalassemia by therapeutic modality

• Gene therapy
• Small molecule
• Protein
• Biological
• Plant derived

According to this pipeline analysis report, the majority of molecules that are currently in the drug development pipeline for Thalassemia are being developed as small molecules. Small molecules are chemically manufactured active substances that can enter cells easily because of their low molecular weight.

Key questions answered in the report include

• What are the drug development molecules in the various development stages for Thalassemia?
• What are the companies that are currently involved in the development of drug development molecules for Thalassemia?
• Insight into discontinued/inactive molecules with appropriate reasoning?
• What are the major regulatory authorities approving drugs in various regions?
• Detailed profiling of each active molecule

Technavio also offers customization on reports based on specific client requirement.