BMS Submits Application for Priority Review of First-in-Class Anemia Therapy Reblozyl in China

Reposted from | Medical Horizon

According to the latest public announcement by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration, two applications for luspatercept–aamt (Reblozyl), an injectable drug from Celgene, a Bristol-Myers Squibb (BMS) company, have been included in the proposed priority review list. The indicated use is for the treatment of adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. Publicly available information indicates that Reblozyl is a first-in-class erythroid maturation agent, representing an innovative class of therapeutics that reduces transfusion burden by modulating late-stage erythroid maturation.

Source: CDE Official Website

According to publicly available information, Reblozyl is a soluble fusion protein composed 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 erythroid cells. It has been granted orphan drug designation, fast track designation, and priority review designation by the U.S. FDA.

In November 2019, the U.S. FDA approved Reblozyl for the treatment of anemia in patients with transfusion-dependent beta-thalassemia. In China, the inclusion of the drug’s application for the indication “adult patients with beta-thalassemia who require regular red blood cell transfusions” in the priority review program means that this innovative therapy is expected to benefit Chinese patients at an accelerated pace.

According to the study results published at the time of Reblozyl’s approval in the United States, data from a Phase 3 clinical trial involving 336 patients with transfusion-dependent β-thalassemia showed that 21% of patients treated with Reblozyl achieved a reduction in transfusion burden of more than 33% compared to baseline, which was significantly superior to the placebo control group (4.5%, p<0.0001).

Notably, in April 2020, the FDA also approved Reblozyl for the treatment of anemia in patients with very low- to intermediate-risk myelodysplastic syndromes (MDS). These patients had failed treatment with erythropoiesis-stimulating agents (ESAs) and required regular red blood cell transfusions to alleviate anemia symptoms. According to the press release, Reblozyl is the first new therapeutic option for this patient population in over a decade.

Public data indicates that anemia is a common condition affecting nearly 25% of the global population. While anemia is transient in most patients, those with certain rare diseases—including beta-thalassemia, myelodysplastic syndromes, and myelofibrosis—may experience long-term chronic anemia. Patients with chronic anemia often rely on frequent red blood cell transfusions to alleviate their symptoms. However, frequent transfusions not only impose lifestyle and financial burdens on patients but also carry risks of adverse effects such as iron overload, infections, and immune reactions.

References:

[1] Center for Drug Evaluation (CDE), National Medical Products Administration of China. Retrieved Jan 22, 2021, from http://www.cde.org.cn/news.do?method=changePage&pageName=service&frameStr=20

[2] FDA approves first therapy to treat patients with rare blood disorder. Retrieved November 8, 2019, from https://www.prnewswire.com/news-releases/fda-approves-first-therapy-to-treat-patients-with-rare-blood-disorder-300954846.html

[3] U.S. Food and Drug Administration (FDA) Approves Reblozyl® (luspatercept-aamt), the First and Only Erythroid Maturation Agent, to Treat Anemia in Adults with Lower-Risk Myelodysplastic Syndromes (MDS). Retrieved 2020-04-04, from https://www.businesswire.com/news/home/20200403005530/en/U.S.-Food-Drug-Administration-FDA-Approves-Reblozyl%C2%AE?from=groupmessage

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