Reblozyl approved for beta thalassaemia treatment

The FDA’s approval of the drug Reblozyl (luspatercept–aamt) to biopharma company Celegene Corporation was awarded after it was granted Fast Track designation, designed to incentivise development of treatments for serious conditions and unmet medical needs.

 “When patients receive multiple blood transfusions, there is a risk (of) iron overload, which can affect many organs,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

“Today’s approval provides patients with a therapy that, for the first time, will help decrease the number of blood transfusions. This approval is an example of our continued progress (with) rare diseases and providing important new drugs to patients earlier.”

Beta thalassaemia, also known as “Cooley’s anaemia,” is an inherited blood disorder that reduces the production of haemoglobin – the protein in red blood cells that contains iron and delivers oxygen to cells throughout the body. Individuals with the condition consequently have low levels of haemoglobin, which results in a lack of oxygen in many parts of the body, leading to weakness, fatigue and more serious complications.

Treatment for people with beta thalassaemia often requires lifelong regimens of chronic blood transfusions for survival and treatment for iron overload due to the transfusions. Such patients are also at an increased risk of developing abnormal blood clots.

The approval of Reblozyl was based on the results of a clinical trial of 336 patients with beta thalassaemia who required RBC transfusions, of which 112 received a placebo. Twenty-one percent of the patients who received Reblozyl achieved at least a 33 percent reduction in transfusions compared to 4.5 percent of the patients who received a placebo. The transfusion reduction meant that the patient needed fewer transfusions over 12 consecutive weeks while taking Reblozyl.

Common side effects for patients taking Reblozyl include headaches, bone pain, arthralgia (joint pain), fatigue, coughs, abdominal pain, diarrhoea and dizziness. Patients may also experience hypertension while using Reblozyl. Health care professionals are advised to monitor a patient’s blood pressure during treatment and check for thrombosis (blood clots).