Luspatercept (Reblozyl®) Becomes First Innovative Therapy for β-Thalassemia Included in China's 2022 National Reimbursement Drug List

Patients' economic pressure and blood transfusion burden are expected to be significantly reduced.

ShanghaiJanuary 18, 2023/PR Newswire/ -- The latest announcement from the National Healthcare Security Administration shows that Bristol-Myers Squibb's globalThe First and Currently Only Erythropoiesis AgentLiblaze^®(General name "Injectable Luspatercept") is nowIncludedNational Basic Medical Insurance, Work-related Injury Insurance, and Maternity Insurance Drug Catalogue (2022)》(hereinafter referred to as "Medical Insurance Catalog") for β-thalassemia (hereinafter referred to as“β-Thalassemia”) Treatment of adult patients. This isThe First ProductIncluded in the National Medical Insurance Catalogβ-Thalassemia Innovative Treatment Drug`, is expected to significantly`Reduce patients' economic pressure and alleviate the burden of blood transfusion., providing more patients with innovative treatment options. The new edition of the medical insurance directory is expected to be implemented on March 1, 2023, and the reimbursement process and time in various regions will be subject to local government announcements.

As globalThe FirstAnd currentlyOnlyErythropoiesis-stimulating agent, Reblozyl^®Granted priority review and approval by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA), it was approved for marketing in China last year.The first targeted β in over a decade-No thalassemiaInnovative drugs for effective hematopoiesis. Unlike traditional blood transfusion and iron chelation therapy, Reblozyl^®By promoting the maturation of late-stage red blood cells, it effectively reduces the transfusion burden in transfusion-dependent β-thalassemia patients while significantly lowering serum ferritin levels, potentially decreasing the risk of organ damage and mortality caused by iron overload. Currently, Reblozyl^®ObtainedInternational Thalassemia Alliance (TIF) and the Chinese Medical Association Hematology BranchWillThe dual recommendation was included in the "2021 Guidelines for the Management of Transfusion-Dependent Thalassemia" and the "Chinese Guidelines for the Diagnosis and Treatment of Transfusion-Dependent β-Thalassemia (2022 Edition)" (Class 1A recommendation), becoming a key reference in this treatment field.New Standard Treatment。

After being included in the medical insurance this time, the long-unmet treatment needs of Chinese β-thalassemia patients are expected to be further improved, which will bring positive significance to alleviating the social blood source shortage, improving patients' quality of life, and saving critical medical resources. This highlights the clinical and social value that innovative drugs bring to patients and society.

Professor Depei Wu, Chairman of the Hematology Society of the Chinese Medical Association and Director of the Department of Hematology at the First Affiliated Hospital of Soochow UniversitySaid: "Thalassemia is a serious disease with high heritability and fatality. Adult patients have long faced problems such as limited treatment options and severe economic burdens. The launch of LuspaterceptEffectively fills the gap in the field of innovative treatments., providing patients with a powerful weapon against diseases. In clinical treatment, maximizing patient benefits can only be achieved by ensuring simultaneous access to ‘availability’ and ‘affordability.’ After Rottestip is included in medical insurance, it will be better...Meet the Long-term Treatment Needs of Patients, significantlyReduce the financial burden on patients and their families"It is expected that policies can be implemented as soon as possible across China, especially in areas with a high incidence of thalassemia, so that more patients can benefit."

β-Thalassemia is a hereditary blood disorder caused by mutations or deletions in the globin peptide chain gene.^[1], the number of β-thalassemia gene carriers worldwide is approximately 800-900 million, accounting for about 1.5% of the global population.^[2]In China, Guangxi, Guangdong, Fujian, Hunan, Yunnan, Guizhou, and Sichuan are regions with a high incidence of thalassemia. As an autosomal recessive genetic disorder, if both parents are carriers of the thalassemia gene, there is approximately a 25% chance that their child will have severe thalassemia. Data shows that Chinese adult patients with transfusion-dependent β-thalassemia account for about 30% of the globally traceable adult patient population.^[3]Compared with thalassemia children, adult patients with severe conditions often face serious challenges in terms of treatment options, treatment adherence, economic burden, and quality of life. Difficulties in blood transfusion, non-standard iron chelation, and heavy economic burdens prevent some patients from completing standardized treatment, leading to the occurrence of complications and a reduction in life expectancy.^[4]. The world's first erythroid maturation agent, Reblozyl^®The approval in China for adult patients with thalassemiaReduce the Burden of Blood Transfusion，Improve Quality of LifeBrings new options. It is estimated that Chinese patients who respond to the treatmentAnnual blood transfusion volume is expected to decrease by approximately57%^[5]，PartHealth-related quality of life scores improved.Costs of complication treatment decreased accordingly. After being included in the medical insurance this time, it is expected to better meet the long-term treatment needs of patients and help more adult patients with β-thalassemia benefit from innovative treatments.。

Ms. Chen Siyuan, Vice President of Bristol-Myers Squibb, General Manager of China and Asian Regional Markets, and President of Bristol-Myers Squibb China, stated,"Bristol-Myers Squibb has been deeply rooted in China for 40 years. We have always focused on critical disease areas with urgent clinical needs that remain unmet, transforming patients' lives through scientific leadership. Thanks to the continuous deepening and reform of the national innovative drug review and approval system and medical insurance policies, the clinical accessibility of innovative drugs has greatly improved. Reblozyl^®Being included in the National Medical Insurance Catalogue only seven months after commercial launch reflects the important role of the Chinese government in promoting innovation and improving patient access, and also demonstrates the country's recognition of the value of innovative drugs. In the future, Bristol-Myers Squibb will continue to honor our commitment by collaborating with payers, clinical experts, and health economists to enhance patient access to innovative drugs within China’s multi-layered medical security system."

About β-Mediterranean Anemia

β-Thalassemia (hereinafter referred to as "β-thal") is a hereditary blood disorder caused by defects in hemoglobin genes. In China, Guangxi, Guangdong, Fujian, Hunan, Yunnan, Guizhou, and Sichuan are regions with high incidence of β-thal.

Ineffective hematopoiesis is one of the key pathophysiologic mechanisms of β-thalassemia, leading to the production of unhealthy red blood cells and a reduction in the number of normal red blood cells, thereby causing severe anemia. The chronic anemic state often leaves patients in a weakened condition and may lead to complications and other serious health issues.

About Reblozyl^®

Reblozyl^®(General name "Injectable Luspatercept") is a globally pioneering erythroid maturation agent. Its unique mechanism of action can regulate late-stage erythrocyte maturation and improve ineffective hematopoiesis. Currently, Reblozyl^®Has been approved for the following indications in multiple countries, including Europe and the United States:

1) Adult patients with β-thalassemia who require regular red blood cell transfusions;*

2) Treatment of anemia in adult patients with very low to intermediate risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T), who have failed erythropoiesis-stimulating agent (ESA) therapy and require transfusion of 2 or more units of red blood cells within 8 weeks.**

* Indications approved in China: For the treatment of patients requiring regular red blood cell transfusions with ≤15 units of red blood cells transfused.^[6]/24-week β-thalassemia adult patients

**This indication has not yet been approved in China.

About Bristol-Myers Squibb China

Bristol-Myers Squibb is a global leading biopharmaceutical company with the mission to "research and provide innovative medicines to help patients overcome serious diseases," always committed to the corporate vision of "leading science, transforming patients' lives." In China, Bristol-Myers Squibb aims to become an innovation leader "rooted in China, originated from China," focusing on accelerating the introduction of innovative drugs in core disease areas such as oncology, hematology, and immunology, to benefit Chinese patients as soon as possible.

Celgene and Juno Therapeutics are wholly-owned subsidiaries of Bristol-Myers Squibb Company. In some markets outside the United States, due to local laws, Celgene and Juno Therapeutics are respectively referred to as Celgene – A Bristol-Myers Squibb Company and Juno Therapeutics – A Bristol-Myers Squibb Company.

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[1] Thalassemia

[2] P1. Colah R, 2010

[3] PubMed, Celgene materials, Orphanet J Rare Dis. 2010; 1. Early launch countries

[4] "Economic Burden Report of Adult Patients with Severe β-Thalassemia in China"

[5] Ali T. Taher, et al. Presented at the 26th EHA, 2021, EP1304

[6] In overseas clinical studies, one unit of red blood cells is 200-350ml of concentrated red blood cells, and should be converted according to clinical practice in China.