BMS Submits NDA for Luspatercept (Reblozyl) in China, Granted Priority Review for Adult Beta-Thalassemia Patients Requiring Regular RBC Transfusions

On February 6, the CDE website showed that Celgene, a subsidiary of Bristol-Myers Squibb, submitted and had accepted for review its marketing application in China for "Luspatercept for Injection." This drug is an erythroid maturation agent used for adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

According to a Bristol-Myers Squibb press release, this is the company’s first innovative hematology drug to be included in the priority review program after submitting overseas clinical trial data to the Center for Drug Evaluation (CDE). The drug was approved for marketing in China by directly leveraging overseas clinical data without conducting domestic clinical trials, thereby setting a new benchmark for the “China speed” of importing new drugs.

Luspatercept for Injection (Luspatercept; brand name in English: Reblozyl; proposed Chinese brand name: Libuluoze) is a first-in-class erythroid maturation agent that modulates late-stage erythroid maturation. Prior to its integration with Bristol-Myers Squibb, Celgene jointly developed Luspatercept with Acceleron.

According to the Insight Global New Drug Database, luspatercept was first approved by the U.S. FDA in November 2019 for the treatment of adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. This indication is the same as the one currently submitted for marketing approval in China. The drug has also been approved in Canada and the European Union. Furthermore, luspatercept has received approval in the United States and the European Union for the treatment of anemia associated with myelodysplastic syndromes (MDS) with ring sideroblasts or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis.

Luspatercept Indications Global Status ▼

From the Insight database (http://db.dxy.cn/v5/home/)

β-thalassemia is a group of hereditary blood disorders characterized by anemia, caused by reduced or absent synthesis of β-globin chains. It is one of the most common hereditary hemoglobin disorders worldwide. The overall prevalence in China is 0.67%, while in high-prevalence regions in southern China, the average prevalence is approximately 2%. Severe β-thalassemia presents as chronic progressive hemolytic anemia, and patients require lifelong blood transfusions, iron chelation therapy, or hematopoietic stem cell transplantation to sustain life.

However, blood shortages and unstable supplies of blood products, coupled with the fact that long-term transfusions can lead to iron overload, increased financial burden, and potential risks of infection, make lifelong transfusion therapy difficult to sustain. There is an urgent need among patients for innovative medications.

As the world’s first recombinant fusion protein therapeutic, luspatercept enhances hemoglobin levels by promoting the maturation of late-stage erythroid cells. Its novel mechanism of action is expected to reduce patients’ reliance on blood transfusions and iron chelation therapy, improve treatment adherence, and mitigate the potential risks associated with repeated transfusions.

The priority review of luspatercept was based on the global Phase III clinical trial named BELIEVE. BELIEVE is a randomized, double-blind, placebo-controlled, multicenter Phase III clinical study that compared the efficacy of luspatercept plus best supportive care (BSC) versus placebo plus BSC in adult patients with β-thalassemia who require regular red blood cell transfusions. The trial enrolled a total of 336 patients across 65 clinical trial centers in 15 countries.

The results showed that after treatment with Luspatercept, 21.4% of patients experienced a reduction in transfusion burden by more than 33% compared to baseline, which was significantly better than the placebo group (4.5%). Additionally, iron overload in patients was significantly reduced, potentially improving their quality of life.

We look forward to a smooth review and approval process for this drug, providing patients in China with new therapeutic options that offer clinical advantages and help improve the quality of life for relevant patients.