As the first and only approved RBC maturing agent in China, Librosze is the first innovative drug approved to treat β-thalassemia in China for more than a decade
Through its unique mechanism of action, Libroze promotes late red blood cell maturation, repairs the patient's own hematopoietic function, and improves ineffective hematopoiesis
The phase III clinical study confirmed that Librosze can not only effectively reduce the transfusion burden in patients with blood transfusion-dependent β-thalassemia, but also significantly reduce the serum ferritin in patients, which may reduce the risk of organ injury and death due to iron overload
Librosze has received Guidelines from the International Federation of The Poor (TIF) for the treatment of transfusion-dependent β-thalassemia in adults
On January 26, 2022, Bristol-Myers Squibb announced that its world's first and currently only red blood cell maturation agent, Libroze (common name "Rotxip for Injection"), has passed the priority review and approval process of the State Drug Administration of China and has been approved for the treatment of adult patients with β-thalassemia (hereinafter referred to as "β-ground poverty") that requires regular infusion of red blood cells and the ≤ of 15 units/24 weeks, becoming the first innovative drug approved to treat poverty in China for more than a decade.
Traditional treatment has limitations, and the local poverty bureau needs to be solved urgently
β-ground poverty is an inherited hemoglobin disorder. Hematopoietic stem cell transplantation is currently the only treatment that can be cured, but there are problems such as low matching probability and age restrictions. Previously, for adult patients who could not receive transplants, they could only rely on lifelong blood transfusions and iron removal to maintain life.
"China's β-poor patients face a severe blood transfusion dilemma. Although the Clinical Practice Guidelines for β-Thalassemia (2020 Edition) clearly stipulate that when patients < Hb 90g/L, the blood transfusion program is initiated every 2-5 weeks, but due to blood pressure, 48.67% of poor patients cannot be treated with blood transfusion on time and on demand. In addition, long-term blood transfusions can lead to iron overload, and patients need to cooperate with iron removal therapy to prevent excess iron deposition in the heart, liver, pancreas and other organs and cause organ damage or even failure. According to the survey, 98% of blood transfusion-dependent patients in China's β-high incidence areas have failed to adhere to iron removal or often miss due to insufficient understanding of the harm of iron overload, inconvenience and its side effects. Professor Wu Depei, chairman of the Hematology Branch of the Chinese Medical Association and director of the Department of Hematology of the First Affiliated Hospital of Soochow University, said, "The approval of Libroze will break the dilemma, bring patients new treatment options other than traditional blood transfusion and iron removal, reduce the transfusion burden of transfusion-dependent β-poor patients, reduce the risk of iron overload caused by blood transfusion, and bring positive social significance to alleviate blood source tension." ”
The approval is based on BELEVE, a randomized, double-blind, placebo-controlled, multicenter phase III clinical study. A total of 336 adult patients with β-ground poverty requiring regular blood transfusions were enrolled in the study, with a baseline transfusion burden of 6 to 20 units of erythrocytes transfused within 24 weeks prior to enrollment, with no period of continuous transfusion beyond 35 days. Patients were randomized on a 2:1 ratio to libroze plus optimal supportive care (including blood transfusion, iron removal, anti-infective therapy, and nutritional support) or placebo plus optimal supportive care.
The primary endpoint results showed that at weeks 13 to 24 of patients in the Libroze group, the transfusion burden decreased by ≥33% (and at least 2 erythrocyte units) from baseline, nearly 5 times higher than in the placebo group (4.5%, p
The first mechanism is precisely applied to significantly improve ineffective hematopoiesis
β-ground poverty is one of the most common hereditary blood disorders characterized by ineffective hematopoiesis, manifested by an insufficiency of normal red blood cells produced by the body's hematopoiesis, leading to anemia. At present, the study has found that the transduction of the TGF-β/Smad2/3 signaling pathway in patients with β-ground poverty can lead to advanced erythrocyte maturation disorders and cause insufficient red blood cell production in patients with β-ground poverty.
Librosze is the world's first and currently the only innovative drug for β-poor advanced erythrocyte maturation disorder. As an artificially designed fusion protein, Libroze acts on the late stages of erythrocyte maturation, and reduces the transduction of the abnormally enhanced Smad2/3 signaling pathway by binding to a specific TGF-β superfamily ligand, a key factor that regulates erythrocyte maturation, thereby restoring advanced erythrocyte maturation and enabling the body to produce more normal red blood cells.
"For a long time, the innovation and development of drugs β-ground poverty has been relatively slow, and no new drugs have appeared in China for more than ten years. Fortunately, with the continuous understanding of the mechanism of ineffective hematopoiesis, the world's first red blood cell maturing agent Libroze has made pioneering progress in this field, and its unique mechanism of action can repair the patient's own hematopoietic function and improve ineffective hematopoiesis. Professor Lai Yongrong, member of the National Standing Committee of the Hematology Branch of the Chinese Medical Association and director of the Department of Hematology of the First Affiliated Hospital of Guangxi Medical University, pointed out, "Libroze is the first truly targeted treatment drug for local poverty in China, which only needs to be injected subcutaneously every three weeks to effectively reduce the incidence of anemia, iron overload and blood transfusion complications, and significantly reduce the time and frequency of patient visits, blood transfusions and iron removal." This will not only bring qualitative improvement to the quality of life of patients, but also greatly promote the standardization process of poverty treatment in China. It is expected that after this approval, Librosze can become a new standard treatment for adult transfusion-dependent β-poor patients in China. ”
Lead science and innovation, "hand" to protect the poor and standardized diagnosis and treatment
Based on the results of the BELEVE study, Librosze has now been included in the 2021 Guidelines for the Administration of Transfusion-Dependent Thalassemia by the International Federation of Poverty (TIF) as the recommended therapy for transfusion-dependent adult β-poor patients.
In addition to accelerating the introduction of innovative drugs to benefit Chinese β-poor patients, Bristol-Myers Squibb is also concerned about the development of standardized diagnosis and treatment of β-local poverty in China. Initiated by Beijing Kangmeng Charity Foundation, supported by Bristol-Myers Squibb and participated by top experts in Chinese hematology, the "Standardized Diagnosis and Treatment of Thalassemia" project has been officially launched during the 4th China International Import Expo in 2021. The project aims to establish the diagnosis and treatment standards of poverty in China, form a hospital alliance in areas with high incidence of poverty, and promote the improvement of the standardized diagnosis and treatment level of poverty; at the same time, explore the clinical application of innovative drugs to bring better life "hand care" to patients.
"Librosse is Bristol-Myers Squibb China's first innovative drug approved for marketing in the field of hematology through overseas research data and priority review and approval procedures, and its approval is the first important milestone in the field of hematology of Bristol-Myers Squibb's 'China 2030 Strategy'. We sincerely thank the Chinese government for its innovative policy support to optimize drug review and approval and accelerate the benefit of urgently needed drugs in clinical practice to Chinese patients." "In Chinese mainland the future, we will actively respond to the attention and appeals of governments at all levels to promote advanced poverty prevention and control models, continue to explore cooperation with the Chinese government, payers and third-party institutions, and improve drug accessibility and change patient lives through innovative access models and diversified initiatives, "In the future, we will actively respond to the attention and appeal of governments at all levels to promote advanced poverty prevention and control models." ”
About β - thalassemia
β-thalassemia (later known as "β-ground poverty") is an inherited blood disorder caused by a defect in the hemoglobin gene. In China, Guangxi, Guangdong, Fujian, Hunan, Yunnan, Guizhou, Sichuan and other places are high incidence areas of β-land poverty.
Ineffective hematopoiesis is an important pathophysiological mechanism of β-ground poverty, which leads to the production of unhealthy red blood cells and a decrease in the number of red blood cells, resulting in severe anemia. Chronic anaemic conditions result in patients who are usually weaker and can cause complications and other serious health problems.
About Librosze
Libroze (generic name "Rotsipro for Injection") is the world's first red blood cell maturation agent, its unique mechanism of action can regulate late red blood cell maturation, improve ineffective hematopoiesis. Currently, Librosze has been approved globally for the following indications:
1) Adults who require regular infusion of red blood cells β- thalassemia;
2) Treatment of very low- to moderate-risk myelodysplastic syndrome with cyclic ferrulocytes (MDS-RS) who failed treatment with erythropoietic stimulants (ESAs) and required infusion of 2 or more units of erythrocytes within 8 weeks, or anemia in adult patients with myelodysplastic syndrome/myeloproliferative tumors with cyclic ferroblasts and thrombocytosis (MDS/MPN-RS-T).
In addition, applications for marketing authorization for libroze's indications for the treatment of adults with non-transfusion-dependent (NTD) β-thalassemia have been submitted in the United States and are eligible for priority review.
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