FDA Approves First Treatment for Rare Blood Disorder, Beta Thalassemia

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.

People with beta thalassemia often have lifelong regimens of chronic blood transfusions in order to survive, and then need treatment for iron overload due to the transfusions.

“When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs,” 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, said in a statement. “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 for rare diseases and providing important new drugs to patients earlier.”

Reblozyl was approved based on a trial of 336 patients, 112 of whom received placebo. The trial showed that 21.0% of the patients receiving Reblozyl achieved at least a 33.0% reduction in transfusions. Patients on placebo only had a 4.5% reduction in transfusions.

The treatment is manufactured by Celgene and Acceleron, and the list price will be $3441 per 25 mg vial, according to Biopharma Dive.

A total of 3.6% of patients experienced serious adverse reactions, the most common of which were headache (26% for Reblozyl vs 24% for placebo), bone pain (20% vs 8%), arthralgia (19% vs 12%), fatigue (14% vs 13%), cough (14% vs 11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%), and dizziness (11% vs 5%).

Nadim Ahmed, president of global hematology and oncology for Celgene called the approval “an important milestone,” since patients with anemia due to beta thalassemia have limited treatment options.

Another treatment in the pipeline for beta thalassemia will be coming from bluebird bio. LentiGlobin will be a gene therapy, which will come with a high price tag. Although the treatment is not yet approved, bluebird bio has already explained that the intrinsic value of the treatment, based on quality of life and life extension, would be $2.1 million. The company has already said it plans to price the treatment south of that number.

In addition, bluebird bio plans to roll out a unique financing mechanism to pay for the gene therapy. The model would spread payments out over 5 years in equal installments. The first installment is the equivalent of an access fee, but the rest of the installments are at risk and are based on the success of the treatment.