BMS Announces CDE Priority Review for Luspatercept in Adult Patients with Transfusion-Dependent Beta-Thalassemia in China

Shanghai, China, February 4, 2021 /PRNewswire/ -- Bristol-Myers Squibb China announced that its marketing application for the erythroid maturation agent luspatercept (English brand name: Reblozyl; proposed Chinese generic name: Luspatercept for Injection; proposed Chinese brand name: Libuluoze) has been formally accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration and granted priority review status, for the treatment of adult patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.

“Since 2018, we have had the privilege of witnessing the continuous breaking of records for ‘China’s New Drug Speed.’ We sincerely thank the Chinese government for optimizing drug review and approval,”Innovative Policy Support to Accelerate the R&D of Clinically Urgent Drugs.”Dr. Xuejun Cai, Vice President of Global R&D and Head of R&D, China, at Bristol-Myers Squibbstated, “Luspatercept is the first innovative hematology drug from Bristol-Myers Squibb China to be included in the priority review process after submitting overseas clinical trial data to the CDE. We believe this innovative drug will fill the gap in the treatment of thalassemia in China and transform patients’ lives.”

Lifelong Blood Transfusions Are Hard to Guarantee; The Dilemma of Thalassemia Treatment Awaits Resolution

β-Thalassemia is a group of hereditary blood disorders characterized by anemia caused by reduced or absent synthesis of β-globin chains.^[1]. Severe β-thalassemia is a rare disease characterized by chronic progressive hemolytic anemia. Patients currently depend on lifelong blood transfusions, iron chelation therapy, or hematopoietic stem cell transplantation to sustain life.

However, in reality, the treatment of severe beta-thalassemia in China is facing multiple challenges. Blood source shortages, unstable supply of blood products, and long-term transfusions that can lead to iron overload, increased economic burden, and potential infection risks make lifelong transfusion therapy difficult to sustain. There is an urgent need for innovative drugs among a large number of patients.

World’s First Mechanism of Action, “Hand” in Hand to Safeguard the Lives of Chinese Patients

The advent of luspatercept is expected to break the therapeutic impasse for β-thalassemia in China. As the world’s first recombinant fusion protein drug, luspatercept enhances hemoglobin levels by promoting the maturation of late-stage erythroid cells.

Its novel mechanism is expected to reduce patients’ reliance on blood transfusions and iron chelation therapy, thereby improving treatment adherence. It also helps avoid the potential risks associated with repeated transfusions, such as long-term iron overload leading to iron deposition in organs like the heart and liver, which can subsequently result in heart failure, liver cirrhosis, and even death.

Based on the global Phase III clinical trial named BELIEVE, which supported the priority review of Luspatercept, 21.4% of patients experienced a reduction in transfusion burden of more than 33% compared to baseline after treatment with Luspatercept.^[2], significantly superior to the placebo group (4.5%), and patients' iron overload was significantly reduced, thereby potentially improving patients' quality of life^[3]。

“The inclusion of luspatercept in the priority review also marks the smooth progress of Bristol-Myers Squibb’s ‘China Strategy 2030.’”Dr. Xuejun Cai, Vice President of Global R&D and Head of R&D, China, at Bristol-Myers Squibbstated, “With the recent official implementation of ‘China Strategy 2030,’ we are committed to becoming a leader in core therapeutic areas such as oncology, hematology, and immunology. Over the next five years, we will accelerate the introduction of nearly 30 innovative drugs or indications.”SymptomMost of them have the potential to become first-in-class or best-in-class products in their respective fields. We will take concrete actions to “lead science and transform patients’ lives,” thereby advancing the implementation of the national strategy “Healthy China 2030.”

AboutLuspatercept

Luspatercept is a first-in-class erythroid maturation agent used to modulate late-stage erythroid maturation. Prior to its integration with Bristol-Myers Squibb, Celgene jointly developed luspatercept with Acceleron.

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 (RBC) transfusions; it has also been approved 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.

AboutBELIEVE Study

BELEVE is a randomized, double-blind, placebo-controlled, multicenter Phase III clinical study comparing 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. The median age of patients in both groups was 30 years. A total of 336 patients were randomized into two groups: one group received luspatercept at a dose of 1.0 mg/kg (224 patients), and the other group received placebo (112 patients), administered via subcutaneous injection every 21 days for 48 weeks. Based on the recommendations of the independent Data Safety Monitoring Board, crossover to luspatercept treatment was permitted after unblinding. Patients receiving luspatercept will be followed up for 3 years. The study was conducted at 65 clinical trial centers across 15 countries.

About β- Thalassemia

β-Thalassemia is one of the most common inherited hemoglobin disorders worldwide, caused by mutations in the β-globin gene that result in the absence or reduced synthesis of this protein during erythropoiesis.

The overall prevalence in China is 0.67%, while in high-prevalence regions in southern China (including Guangdong, Guangxi, Fujian, Hunan, Yunnan, Guizhou, and Sichuan), the average prevalence is approximately 2%. Clinically, β-thalassemia is classified into minor, intermedia, and major forms based on disease severity. Individuals with thalassemia minor are generally asymptomatic or exhibit mild anemia, making the condition easily overlooked or detected only during family screening. In contrast, thalassemia major presents as chronic progressive hemolytic anemia, severely impacting patients' quality of life and even survival.

[1] Hu Zhangxue et al., Construction of a diagnostic gene chip for β-thalassemia and congenital achondroplasia. Journal of Third Military Medical University, 2008, 30(3)

[2] Celgene Corporation and Acceleron Pharma Announce Results of the Phase 3 BELIEVE Trial Evaluating Luspatercept in Adult Patients with Beta-Thalassemia at ASH 2018. Retrieved December 3, 2018, from https://ir.celgene.com/press-releases/press-release-details/2018/Celgene-Corporation-and-Acceleron-Pharma-Announce-Results-of-the-Phase-3-BELIEVE-Trial-Evaluating-Luspatercept-in-Adult-Patients-with-Beta-Thalassemia-at-ASH-2018/default.aspx

[3] Cappellini M D , Taher A T , Piga A , et al. Health-Related Quality of Life Outcomes for Patients with Transfusion-Dependent Beta-Thalassemia Treated with Luspatercept in the Believe Trial[J]. Blood, 2020, 136(Supplement 1):8-9.