CHEARS

Beta thalassemia is a blood disorder that reduces the production of hemoglobin which is the iron-containing protein in red blood cells that carries oxygen to cells in the body. In people with beta thalassemia, due to low levels of hemoglobin there will be a lack of oxygen in the body.

Approximately 1–5% of the global population are carriers for a genetic thalassemia mutation. Individuals with beta thalassemia minor have a mutation in one HBB gene, while individuals with the intermediate and major forms have mutations in both HBB genes. Symptoms of Beta Thalassemia includes tiredness, shortness of breath, a fast heartbeat pale skin, yellow skin and eyes (jaundice), moodiness and slow growth.

Patients with transfusion-dependent beta thalassemia, require lifelong management with red blood cell (RBC) transfusions, in order to survive, and then need treatment for iron overload due to the transfusions which can affect many organs further results in a substantial cost and burden to patients. The only curative treatment for patients with transfusion-dependent beta thalassemia is allogeneic hematopoietic cell transplantation (alloHCT), which also is associated with a variety of adverse events. Finding new treatment options for these patients is a high priority for the hematology community.

Treatment with luspatercept alleviated patient’s transfusion burden, with approximately 70% of patients achieving a clinically significant reduction in RBC transfusion requirements. Adult patients with beta thalassemia will now have an FDA-approved treatment available with luspatercept-aamt (Reblozyl). The therapy treats the rare inherited blood disorder, which requires patients to have regular red blood cell transfusions. Common adverse events associated with luspatercept-aamt includes headache, bone pain, joint pain, fatigue, cough, abdominal pain, diarrhea and dizziness

This approval is an example of our continued progress for rare diseases and providing important new drugs to patients, to make them believe that their condition is being managed effectively.