FDA Approves Lusaptercept-aamt for Anemia in Patients with Beta Thalassemia

The United States Food and Drug Administration (FDA) has approved luspatercept-aamt (Reblozyl) for the treatment of anemia in adult patients with beta thalassemia who require regular blood red blood cell transfusions.

The approval, which was awarded to the Celgene Corporation, makes lusaptercept-aamt the first approved treatment for patients with the rare blood disorder.

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

An inherited blood disorder, beta thalassemia—or Cooley’s anemia—reduces the production of hemoglobin in a patient’s blood. As a result, many patients undergo supportive treatment that includes the need for lifelong chronic blood transfusions. Additionally, these patients are also at an increased risk of developing abnormal blood clots.

The application for luspatercept-aamt had previously received Fast Track designation from the FDA and the therapy also received an Orphan Drug designation.

The FDA’s approval of luspatercept-aamt is based off the results of a phase 3, randomized, double-blind, placebo-controlled, multi center BELIEVE trial, which evaluated the therapy’s efficacy and safety in more than 300 patients. Results fo the trial revealed 21% of patients receiving luspatercept-aamt experienced a reduction in transfusions of 33% or more compared to just 4.5% of patients receiving placebo.

The most common adverse reactions were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea, and dizziness. A release from Celgene Corporation and Acceleron Pharma Inc. noted 3.6% of patients treated with luspatercept-aamt experienced thromboembolic events, including deep vein thromboses, pulmonary embolus, portal vein thrombosis, and ischemic stroke.

Celgene Corporation announced it expects luspatercept-aamt to be available within 1 week of the FDA approval on November 8.

“We’re thrilled that Acceleron’s first approved medicine is one with the potential to help patients with beta thalassemia, who have been in need of new treatments for this lifelong disease,” said Habib Dable, president and chief executive officer of Acceleron.