Sickle Cell Anemia - A Pipeline Analysis Report

Overview of the drug development pipeline for sickle cell anemia

It has been observed that sickle cell anemia is a disease characterized by the production of abnormal hemoglobin. It binds with other abnormal hemoglobin molecules within the red blood cells and leads to rigid deformation of the cell. This deformation inhibits the ability of the cell to pass through small vascular channels. This may result in sludging and congestion of vascular beds, followed by tissue ischemia and infarction. Infarction is common throughout the body in a patient affected by sickle cell anemia and is responsible for the earliest clinical manifestation and acute pain crisis. Sickle cell anemia generally begins at around five to six months of age. The common symptoms of sickle cell anemia are extreme fatigue, fussiness in babies, bedwetting, and swelling of the hands and feet. It is estimated that about 75% of cases of sickle cell anemia occur in Africa. Monotherapy is emerging as the most efficient approach to the treatment of sickle cell anemia.

According to this pipeline analysis report, all the drug molecules in the pipeline are being developed for sickle cell anemia. Our market research analysts have also identified that most of these molecules are in the pre-clinical development stage and a considerable number of molecules have been discontinued from development.

Drug development pipeline report for sickle cell anemia: 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 sickle cell anemia. 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 molecules discontinued by companies.

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

• ArQule
• AstraZeneca
• Hillhurst Biopharmaceuticals

Therapeutic assessment of the drug development pipeline for sickle cell anemia by route of administration

• Oral
• Intravenous
• Implant
• Subcutaneous
• Undisclosed

The oral route of administration (ROA) involves the delivery of the drug directly into the mouth cavity and in the intravenous technique, the liquid drug molecules are administered into a vein, which will have a more direct effect on the target cells.

Therapeutic assessment of the drug development pipeline for sickle cell anemia by therapy

• Monotherapy
• Undisclosed

According to this pipeline analysis report, most of the molecules that are currently in the drug pipeline for sickle cell anemia are being developed as monotherapy drugs and most of these molecules are in the pre-clinical stage of development.

Key questions answered in the report include

• What are the gene therapy molecules in the various development stages for sickle cell anemia?
• What are the companies that are currently involved in the development of drug molecules for sickle cell anemia?
• 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.