FDA Approves Luspaterecpt-aamt for Anemia

November 12, 2019

Shown to reduce regular RBC transfusions in adults with beta thalassemia.

The FDA has approved luspaterecpt-aamt (Rebloyzl, Celgene) for the treatment of anemia in adult patients with beta thalassemia (a rare, inherited blood disorder caused by genetically defunct hemoglobin) who require regular RBC transfusions. 

Luspaterecpt-aamt is currently the first and only erythroid maturation agent approved by the FDA, according to Celgene’s official release, thus representing a new class of drug therapy for patients with anemia. The drug is not indicated as a substitute for RBC transfusions in patients who require immediate correction of anemia. 

“There are very limited options for patients living with anemia due to beta thalassemia who are dependent on long-term red blood cell transfusions,” said Nadim Ahmed, president of global hematology and oncology for Celgene in a statement. “We are pleased to make REBLOZYL available as a new therapy for these patients to help address their anemia, a significant clinical complication of beta thalassemia.”

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Approval is based upon results BELIEVE-a phase 3, randomized, double-blind, placebo-controlled, multi-center trial evaluating the safety and efficacy of luspaterecpt-aamt in treating anemia in adult patients with beta thalassemia who require between 6 and 30 RBC transfusion units per 24 weeks, with no transfusion-free period greater than 35 days during that period.

Overall, the trial returned clinically meaningful and statistically significant improvement in primary and secondary endpoints, according to an official release. 

Primary Endpoint: At least a 33% reduction (or at least 2 units) in RBC transfusion burden between weeks 13-24 after randomization. 

  • Luspaterecpt-aamt arm: 21.4% of patients

  • Placebo: 4.5% of patients

Secondary Endpoint: 33% reduction (at least 2 units) of RBC transfusion burden between weeks 37-48 after randomization. 

  • Luspaterecpt-aamt arm: 19.6% of patients

  • Placebo: 3.5% of patients

Efficacy Endpoint: At least 50% burden reduction during weeks 13-14 and 37-48 after randomization

  • Luspaterecpt-aamt: 7.6% of patients (weeks 13-24); 10.3% (weeks 13-24)

  • Placebo: 1.8% (weeks 37-48); 0.9% (weeks 37-48)

Adverse events reported throughout the trial include thromboembolic events, including: deep vein thromboses, pulmonary embolus, portal vein thrombosis, and ischemic stroke, (3.6% of patients); and hypertension (10.7% of patients). 

Serious adverse events included cerebrovascular accident and deep vein thrombosis; one death due to unconfirmed case of acute myeloid leukemia (AML). 

Permanent discontinuation occurred in 5.4% of patients due to arthralgia, back pain, bone pain, and headache.

Dosage interruptions occurred in 15.2% of patients due to upper respiratory tract infection, alanine aminotransferase increase, and cough.

Warnings and precautions issued with the use of luspaterecpt-aamt include thrombosis/thromboembolism, hypertensions, and embryo-fetal toxicity. 

Other adverse reactions in the prescribing information for luspaterecpt-aamt include: headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea, and dizziness. 

 

Full Prescribing Information