BMS’s First-in-Class Anemia Therapy Luspatercept Nears Approval in China for Adult Beta-Thalassemia Patients

Source: Medicinal Landscape

The latest announcement on the official website of China’s National Medical Products Administration (NMPA) shows that the marketing application status of luspatercept for injection, submitted by Celgene Corporation under Bristol-Myers Squibb (BMS), has been updated to "under review." This indicates that the drug is expected to be approved in China soon. Public information reveals that luspatercept is a “first-in-class” erythroid maturation agent, and the indication applied for in this submission is: for the treatment of adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

Screenshot source: NMPA official website

β-Thalassemia is a hereditary blood disorder characterized by anemia, caused by reduced or absent synthesis of the β-globin peptide chain. Luspatercept is a soluble fusion protein consisting of the Fc domain of human immunoglobulin G1 (IgG1) fused to the extracellular domain of activin receptor IIB (ActRIIB). It functions as a ligand trap for TGF-β, preventing the activation of the Smad2/3 signaling pathway, thereby promoting the differentiation and maturation of late-stage erythrocytes and increasing hemoglobin levels.

Currently, Luspatercept has been approved in the United States, Canada, and the European Union for the treatment of adult patients with β-thalassemia who require regular red blood cell transfusions, and in the United States and the European Union for the treatment of anemia in patients with certain blood disorders. Additionally, a supplemental Biologics License Application (sBLA) for Luspatercept for the treatment of adult patients with non-transfusion-dependent β-thalassemia has recently been granted Priority Review designation by the FDA.

In February 2021, the Center for Drug Evaluation (CDE) of the China National Medical Products Administration accepted the marketing application for Luspatercept for injection and included it in the priority review as a "drug eligible for conditional approval" to treat adult patients with β-thalassemia who require regular red blood cell (RBC) transfusions.

According to an earlier press release from Bristol-Myers Squibb, the inclusion of luspatercept's marketing application in China into the priority review is based on a global Phase 3 clinical trial named BELIEVE. This trial is a randomized, double-blind, placebo-controlled, multi-center study conducted across 65 clinical trial sites in 15 countries. The study compared the efficacy of luspatercept plus Best Supportive Care (BSC) versus placebo plus BSC in adult patients with beta-thalassemia requiring regular red blood cell transfusions. Patients receiving luspatercept will be followed up for three years.

BELIEVE clinical study results showed that: after treatment with Luspatercept, 21.4% of patients had a reduction in transfusion burden of more than 33% compared to baseline, significantly better than the placebo group (4.5%). Additionally, patient iron overload was significantly reduced.

Note: The original text has been abridged.

References:

[1] Progress Query of Drug Registration in China National Medical Products Administration. Retrieved Jan 24, 2022, from http://sq.nmpa.gov.cn/datasearch_nmpa/schedule/search.jsp?tableId=43&tableName=TABLE43&columnName=COLUMN464,COLUMN475&title1=Progress Query of Drug Registration

[2] Bristol-Myers Squibb's Innovative Hematology Drug Luspatercept Granted Priority Review by CDE. Retrieved Feb 4, 2021, from https://www.prnasia.com/story/307816-1.shtml

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