FDA approves first treatment for rare blood disorder

The FDA approved Nov. 8 Celegene's drug, Reblozyl, designed to treat anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.

Beta thalassemia, also called "Cooley's anemia," is a rare, inherited blood disorder that reduces the production of hemoglobin, which can lead to a lack of oxygen in many parts of the body and anemia, which can cause pale skin, weakness, fatigue and more serious complications.

Treatment for beta thalassemia often consists of chronic blood transfusions for survival and treatment for iron overload due to transfusions, which can damage organs.

Reblozyl decreases the number of transfusions beta thalassemia patients need.

In the clinical trial of 336 patients, 21 percent who received Reblozyl achieved at least a 33 percent reduction in transfusions, compared to 4.5 percent in patients who took the placebo. The reduction meant patients needed fewer transfusions over 12 consecutive weeks while taking Reblozyl.

Common side effects of the drug include headache, bone and joint pain, fatigue, cough, abdominal pain, diarrhea and dizziness. Patients may also experience hypertension, and healthcare providers are advised to monitor patients' blood pressure during treatment. Patients should also be monitored for blood clots.

Women who are pregnant or breastfeeding should not take Reblozyl, as it may cause harm to a developing fetus or newborn baby.

The FDA granted Reblozyl fast track designation and orphan drug status.

Read the full news release here.

 

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