BMS Announces China Approval of Luspatercept, a First-in-Class Erythroid Maturation Agent for Adult Patients with β-Thalassemia Requiring Regular Red Blood Cell Transfusions

By Medicine View

China's National Medical Products Administration (NMPA) recently announced on its official website that luspatercept, an injectable drug submitted by Celgene Corporation under Bristol-Myers Squibb (BMS), has been approved in China. Public information indicates that luspatercept is a "first-in-class" erythroid maturation agent, and the approved indication in China 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 type of hereditary blood disorder characterized by anemia, caused by reduced or absent synthesis of the β-globin peptide chain. Severe β-thalassemia is a rare disease that manifests as chronic progressive hemolytic anemia. Currently, patients rely on lifelong blood transfusions, iron chelation therapy, or hematopoietic stem cell transplantation to sustain life, and there is an urgent need for new treatment options.

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 TGF-β from activating the Smad2/3 signaling pathway, thereby promoting the differentiation and maturation of late-stage erythrocytes and increasing hemoglobin levels.

▲Luspatercept can increase the hemoglobin content in patients' blood and promote the differentiation of mature red blood cells (Image source: Reference [3]).

According to the press release previously issued by Bristol-Myers Squibb, the novel mechanism of Luspatercept (罗特西普) is expected to reduce patients' reliance on blood transfusions and iron chelation therapy, improve treatment adherence, and also avoid potential risks associated with repeated blood transfusions.

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, the supplemental Biologics License Application 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.

Screenshot source: CDE

According to a press release previously issued by 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 randomized, double-blind, placebo-controlled, multi-center study was 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 who require 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%), and patients had a significant reduction in iron overload.

References:

[1] Information on Pending Drug Approval Documents Released on January 26, 2022. Retrieved Jan 26, 2022, from https://www.nmpa.gov.cn/zwfw/sdxx/sdxxyp/yppjfb/20220126102728166.html

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

[3]Reblozyl (luspatercept-aamt) website. From https://www.reblozyl.com/about-reblozyl-bt/

▽Follow 【WuXi AppTecGermany】WeChat Official Account