BMS Announces First-in-Nearly-20-Years Breakthrough: Reblozyl Approved in China for New Indication in Lower-Risk MDS-Associated Anemia

On June 4, BMS announced its injectable drug Luspatercept(Product name: Reblozyl)Approved for new indications in China,Treatment of anemia caused by very low-risk, low-risk, and intermediate-risk myelodysplastic syndromes requiring regular red blood cell transfusionsAdult patients.

The press release pointed out that this isThe First in Nearly 20 YearsApproved for the treatment ofLower-risk myelodysplastic syndrome(MDS) AnemiaThe innovative drug has the potential to redefine anemia treatment for lower-risk MDS patients who require regular blood transfusions.

Screenshot source: Official WeChat account of the company

Myelodysplastic Syndromes（MDS）It is a heterogeneous myeloid neoplasm originating from hematopoietic stem cells, mainly characterized by peripheral blood cytopenia of one or more lineages caused by abnormal development and ineffective hematopoiesis of bone marrow cells. Clinically, about 77% of patients are diagnosed with lower-risk MDS.（LR-MDS）For these patients, chronic anemia and transfusion dependence are the main clinical issues.

Currently,LR-MDSThe treatment faces many challenges. The clinical application effects of various conventional and supportive treatment methods, such as erythropoietin, immunosuppressants, demethylation drugs, lenalidomide, thalidomide, and androgens, are not ideal and may be accompanied by significant side effects or high costs.

Luspatercept（luspatercept-aamt，Reblozyl）AsWorld's FirstAn erythroid maturation agent. As a ligand trap, luspatercept targets and binds to specific ligands of the transforming growth factor (TGF)-β superfamily that regulate late-stage erythropoiesis, reducing the activation of the Smad2/3 signaling pathway, improving ineffective erythropoiesis, promoting the maturation of late-stage erythrocytes, and increasing hemoglobin levels.

Screenshot source: Corporate official website

Currently,Luspatercept has been approved in the United States and many other countries around the world for β-thalassemia and myelodysplastic syndromes. (MDS) 。In January 2022, the drug was approved in China for the treatment of adult patients with β-thalassemia who require regular red blood cell transfusions and have red blood cell transfusions ≤15 units/24 weeks.

Luspatercept was initially developed by Acceleron Pharma. In August 2011, Celgene(Later acquired by BMS)Collaborated with Acceleron to jointly oversee the development, manufacturing, and commercialization of Reblozyl. In November 2021, Merck acquired$11.5 billionAcquisition of Acceleron Pharma yields luspatercept.

According to BMS financial reports, the sales of Luspatercept have been increasing year by year in recent years, with sales exceeding 1 billion US dollars in 2023, and the total sales for 2024 reached$1.773 billion。

Screenshot source: Insight database

Announced at the 2024 EHA ConferenceBridging Clinical Results of Luspatercept in Chinese Patients（MEDALIST Study Bridging Study MDS-004），This study aims to evaluate the efficacy and safety of luspatercept in treating anemia in Asian patients, including Chinese patients, with lower-risk MDS with ring sideroblasts who are transfusion-dependent, have a heavier baseline, and higher EPO levels.

Data show that: in the ITT population, during weeks 1-24,60% of patients with RBC-TI(8-week RBC Infusion Independence)≥8 weeks, reaching the primary endpointFor patients with RBC-TI≥8 weeks, the median duration of response can reach 24.4 weeks, and 43% of patients achieved RBC-TI ≥12 weeks, with hematological improvement-erythroid response.（mHI-E）The rate was 63%, and the overall efficacy data were basically consistent with the MEDALIST study data. In terms of safety, no new safety events were found in the study.

Screenshot source: Insight database

The conference also announced the use of Lusutrombopag for first-line treatment without erythropoiesis-stimulating agents.（ESA）Latest data from the COMMANDS study on lower-risk MDS treatment.

• The results showed that the luspatercept groupThe treatment response rate and median duration of response were both superior to those in the ESA group., the proportion of patients achieving RBC-TI ≥12 weeks and an average Hb level increase of ≥1.5 g/dL was significantly improved.（70.9% vs 43.1%）；

• Luspatercept GroupRBC-TI≥12 weeks response rate and ≥24 weeks response rate reached 76.4% and 65.9%, respectively., while in the epoetin alfa treatment group, only 55.8% and 45.3%, respectively;

• In terms of reducing red blood cell transfusion volume by ≥50% for a duration of ≥12 weeks, luspatercept compared to ESAMore effective（83.0% vs 66.9%，p=0.0002），The median duration of response was also longer.(130.0 weeks vs 77.0 weeks, p=0.0009).

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