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Introduction β-thalassemia (hereinafter referred to as "β-thalassemia") is a hereditary disease of the blood system with severe symptoms and serious impairment of the quality of life of patients
.
The southeastern coast of China is a high-incidence area of β-thalassemia, with an average prevalence of 2%1
.
At present, the traditional treatment of β-thalassemia in China is facing many difficulties.
Many problems are like shackles that shackle the clinical development.
The demand for innovative drug therapy by the majority of patients is increasing
.
Surprisingly, on January 26, 2022, the world's first and currently only red blood cell maturation agent, Librozer (generic name: rottercept for injection), was approved by the Food and Drug Administration's priority review and approval process.
Approved therapy for adult patients with beta-thalassaemia requiring regular red blood cell transfusions and red blood cell transfusions ≤15 units/24 weeks
.
As the first innovative drug approved in China for the treatment of β-thalassaemia in more than ten years, what is the innovation of Librose? What are the benefits for the treatment of β-thalassaemia in China? This article will focus on you
.
The "Legend" edge out of the sheath finally broke through the transfusion-dependent (TD) β-thalassaemia treatment for a long time limited to hematopoietic stem cell transplantation and symptomatic treatment (mainly lifelong blood transfusion and iron removal), and has been approved and marketed in China since 2000.
The drugs are all iron chelators, and since 2010, no related new drugs have been approved in China
.
For adult TD β-thalassemia patients who are not suitable for transplantation, the existing treatment model faces multiple limitations, such as blood source stress, iron overload caused by blood transfusion, and long-term blood transfusion and iron removal therapy have a great impact on the quality of life.
The clinical needs need to be met urgently.
The dilemma needs to be solved urgently
.
Figure 1.
List of domestically approved drugs for the treatment of blood transfusion-dependent β-thalassaemia since 2000 FDA) approved for the treatment of adult patients with TD β-thalassaemia 2
.
In 2021, Librozer was recommended by the International Thalassaemia Federation (TIF) guidelines for the treatment of transfusion-dependent β-thalassemia in adults 3
.
This time, Librose was quickly approved in China, which is also widely expected
.
In February 2021, Rottecept was granted priority review status by the Center for Drug Evaluation (CDE) of the State Drug Administration through overseas clinical research data, and was approved one year later
.
As an original and innovative drug, rotacept can not only fill the gap in the field of β-thalassaemia treatment in China, but is even expected to break through the current treatment dilemma and open a new chapter in the treatment of β-thalassaemia
.
The new mechanism of removing the old "cloth" is the first to know the truth, and it is also necessary to know the reason
.
Librozer has injected a long-lost "fresh blood" into the field of β-thalassaemia, and its superior efficacy benefits from its unique mechanism of action
.
One of the main disease characteristics of β-thalassemia is ineffective hematopoiesis
.
The current study found that in diseases involving ineffective hematopoiesis, including β-thalassaemia and myelodysplastic syndromes, abnormally enhanced transduction of the transforming growth factor (TGF)-β/Smad 2/3 signaling pathway in patients may be a Inherent features that lead to late erythrocyte maturation disorders, which in turn result in insufficient normal erythropoiesis 4
.
Figure 2.
The generation of β-thalassemia ineffective hematopoiesis and the mechanism of action of rotercept is different from the traditional treatment mechanism.
Libuloze can precisely target and repair patients' advanced erythropoiesis disorders and improve ineffective hematopoiesis
.
Its structure is a novel fusion protein, which can reduce the abnormally enhanced Smad 2/3 signaling pathway by binding to the key cytokines regulating erythrocyte maturation-specific TGF-β superfamily ligands, thereby restoring late erythrocyte maturation and enabling The body can produce more normal red blood cells5, which in turn reduces the amount of blood transfusion required, which may reduce the risk of complications from iron overload and improve the patient's quality of life
.
Based on the above mechanism, Libuloze has been approved globally to treat a variety of ineffective hematopoietic diseases, including β-thalassaemia and myelodysplastic syndrome (MDS)
.
The data is "low" and the multiple benefit mechanism is the cause, and the curative effect is the effect
.
The approval of a new mechanism drug requires a thousand trials and tribulations in clinical trials
.
Librose's approval is based on the randomized, double-blind Phase III study BELIEVE6
.
It compared the efficacy and safety of rotaxip + best supportive care (BSC) with placebo + BSC in adult patients with beta-thalassaemia requiring regular blood transfusions
.
The primary endpoint of the study showed that between weeks 13 and 24, the proportion of patients in the rotercept group with a ≥33% reduction in transfusion burden (and at least 2 units of red blood cells) was nearly 5 times higher than in the placebo group (21.
0% vs.
4.
5%).
, p<0.
0001)
.
Additional analyses showed that, over any 24-week period, the proportion of patients in the rotercept group had a 15-fold reduction in transfusion burden compared with the placebo group (41.
1% vs.
2.
7%); Ferritin levels decreased by 248 μg/L from baseline (increased by 107 μg/L in the placebo group)
.
Subsequently, a post hoc analysis released at the 2021 European Hematology Association (EHA) Congress showed that even in patients who did not meet the primary endpoint at 24 weeks of rotercept, most patients could still benefit from continued treatment after 48 weeks of treatment.
The benefits include the reduction of blood transfusion volume and frequency, as well as the reduction of serum ferritin levels; and the follow-up data show that the blood transfusion burden of patients receiving long-term rotercept continued to decrease
.
Figure 3.
The reduction of blood transfusion burden between the rotercept treatment group and the placebo group In conclusion, the BELIEVE study confirmed that rotercept not only effectively reduces the blood transfusion burden in patients with transfusion-dependent β-thalassaemia, but also significantly reduces serum ferritin, which may It reduces the risk of organ damage and death caused by iron overload, and the benefits are long-term.
At the same time, the adverse events are within the acceptable range, and most of the adverse events are below grade 3 and are well tolerated
.
"Ze" Yao Xinsheng writes a new chapter The excellent curative effect of rotacept in international clinical research is obvious to all, and as an innovative drug just launched in China, the application value and clinical significance of rotacept in China are self-evident
.
As a subcutaneous injection drug, Libuloze only needs to be injected once every 3 weeks, which helps to reduce the frequency and time of medical treatment, blood transfusion and iron removal in patients with β-thalassemia, and effectively reduces the complications caused by blood transfusion and iron removal.
Physical, psychological and economic burden, greatly improve the quality of life of patients
.
In addition, for domestic clinical practice, the drug provides a brand-new TD β-thalassaemia treatment plan, filling the gap in the treatment field, and will also contribute to the implementation and practice of standardized diagnosis and treatment of thalassemia
.
In addition to TD β-thalassaemia, a breakthrough has been made in the application of red blood cell maturation agents in adult patients with non-transfusion-dependent (NTD) β-thalassemia
.
The results of the Phase II BEYOND study7 in patients with NTD β-thalassaemia were presented at the 2021 EHA Congress as LBA (Late-breaking Abstract), and 77.
1% of patients in the experimental group had higher hemoglobin levels during weeks 13-24 without blood transfusions.
Baseline increase of ≥1.
0 g/dL, while no patients in the placebo group met this primary endpoint (p<0.
0001); 89.
6% of patients in the trial group remained transfusion-free from 1 to 24 weeks versus 67.
3% in the placebo group (p=0.
0013)
.
Click to read: Professor Lai Yongrong's in-depth interpretation of the BEYOND study Based on this study, the marketing application of Librozer for the treatment of patients with NTD β-thalassaemia has been granted priority review status by the FDA
.
This also means that rottercept may soon benefit all beta-thalassemia patients
.
During the launch of Rottercept in China, it has witnessed the unremitting efforts of thousands of clinical, scientific research and related staff and the high hopes of many patients
.
The unique mechanism, excellent curative effect and huge potential constitute a solid "foundation" for rotacept in the field of β-thalassaemia treatment.
Build a building for innovative treatment of β-thalassemia in China to shield more β-thalassemia patients from wind and rain; at the same time, it helps to relieve the shortage of blood products in clinical practice and promote the balance of supply and demand of blood resources in the whole society
.
References: [1] Hematology Group of Pediatrics Branch of Chinese Medical Association, Editorial Board of "Chinese Journal of Pediatrics".
Guidelines for the diagnosis and treatment of β-thalassemia major (2017 edition) [J].
Chinese Journal of Pediatrics, 2018, 56(10 ): 6.
[2]https:// Xiangmin, Wang Lin, Yin Xiaolin, et al.
Operational Guidelines for Thalassaemia Prevention and Control[J ].
People’s Military Medical Press, 2011.
[5]Guerra A, Oikonomidou PR, Sinha S, et al.
Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia.
Blood 2019 ;134:568-572.
[6]MD Cappellini, Viprakasit V, Taher AT, et al.
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia[J].
New England Journal of Medicine, 2020, 382 (13):1219-1231.
[7]Taher AT, Cappellini MD, Kattamis A et al.
The BEYOND study: results of a phase 2, double-blind, randomized, placebo-controlled multicenter study of luspatercept in adult patients with non -transfusion-dependent β-thalassemia.
2021 EHA, Abstract: S101.
Click "read the original text", we will make progress together
.
The southeastern coast of China is a high-incidence area of β-thalassemia, with an average prevalence of 2%1
.
At present, the traditional treatment of β-thalassemia in China is facing many difficulties.
Many problems are like shackles that shackle the clinical development.
The demand for innovative drug therapy by the majority of patients is increasing
.
Surprisingly, on January 26, 2022, the world's first and currently only red blood cell maturation agent, Librozer (generic name: rottercept for injection), was approved by the Food and Drug Administration's priority review and approval process.
Approved therapy for adult patients with beta-thalassaemia requiring regular red blood cell transfusions and red blood cell transfusions ≤15 units/24 weeks
.
As the first innovative drug approved in China for the treatment of β-thalassaemia in more than ten years, what is the innovation of Librose? What are the benefits for the treatment of β-thalassaemia in China? This article will focus on you
.
The "Legend" edge out of the sheath finally broke through the transfusion-dependent (TD) β-thalassaemia treatment for a long time limited to hematopoietic stem cell transplantation and symptomatic treatment (mainly lifelong blood transfusion and iron removal), and has been approved and marketed in China since 2000.
The drugs are all iron chelators, and since 2010, no related new drugs have been approved in China
.
For adult TD β-thalassemia patients who are not suitable for transplantation, the existing treatment model faces multiple limitations, such as blood source stress, iron overload caused by blood transfusion, and long-term blood transfusion and iron removal therapy have a great impact on the quality of life.
The clinical needs need to be met urgently.
The dilemma needs to be solved urgently
.
Figure 1.
List of domestically approved drugs for the treatment of blood transfusion-dependent β-thalassaemia since 2000 FDA) approved for the treatment of adult patients with TD β-thalassaemia 2
.
In 2021, Librozer was recommended by the International Thalassaemia Federation (TIF) guidelines for the treatment of transfusion-dependent β-thalassemia in adults 3
.
This time, Librose was quickly approved in China, which is also widely expected
.
In February 2021, Rottecept was granted priority review status by the Center for Drug Evaluation (CDE) of the State Drug Administration through overseas clinical research data, and was approved one year later
.
As an original and innovative drug, rotacept can not only fill the gap in the field of β-thalassaemia treatment in China, but is even expected to break through the current treatment dilemma and open a new chapter in the treatment of β-thalassaemia
.
The new mechanism of removing the old "cloth" is the first to know the truth, and it is also necessary to know the reason
.
Librozer has injected a long-lost "fresh blood" into the field of β-thalassaemia, and its superior efficacy benefits from its unique mechanism of action
.
One of the main disease characteristics of β-thalassemia is ineffective hematopoiesis
.
The current study found that in diseases involving ineffective hematopoiesis, including β-thalassaemia and myelodysplastic syndromes, abnormally enhanced transduction of the transforming growth factor (TGF)-β/Smad 2/3 signaling pathway in patients may be a Inherent features that lead to late erythrocyte maturation disorders, which in turn result in insufficient normal erythropoiesis 4
.
Figure 2.
The generation of β-thalassemia ineffective hematopoiesis and the mechanism of action of rotercept is different from the traditional treatment mechanism.
Libuloze can precisely target and repair patients' advanced erythropoiesis disorders and improve ineffective hematopoiesis
.
Its structure is a novel fusion protein, which can reduce the abnormally enhanced Smad 2/3 signaling pathway by binding to the key cytokines regulating erythrocyte maturation-specific TGF-β superfamily ligands, thereby restoring late erythrocyte maturation and enabling The body can produce more normal red blood cells5, which in turn reduces the amount of blood transfusion required, which may reduce the risk of complications from iron overload and improve the patient's quality of life
.
Based on the above mechanism, Libuloze has been approved globally to treat a variety of ineffective hematopoietic diseases, including β-thalassaemia and myelodysplastic syndrome (MDS)
.
The data is "low" and the multiple benefit mechanism is the cause, and the curative effect is the effect
.
The approval of a new mechanism drug requires a thousand trials and tribulations in clinical trials
.
Librose's approval is based on the randomized, double-blind Phase III study BELIEVE6
.
It compared the efficacy and safety of rotaxip + best supportive care (BSC) with placebo + BSC in adult patients with beta-thalassaemia requiring regular blood transfusions
.
The primary endpoint of the study showed that between weeks 13 and 24, the proportion of patients in the rotercept group with a ≥33% reduction in transfusion burden (and at least 2 units of red blood cells) was nearly 5 times higher than in the placebo group (21.
0% vs.
4.
5%).
, p<0.
0001)
.
Additional analyses showed that, over any 24-week period, the proportion of patients in the rotercept group had a 15-fold reduction in transfusion burden compared with the placebo group (41.
1% vs.
2.
7%); Ferritin levels decreased by 248 μg/L from baseline (increased by 107 μg/L in the placebo group)
.
Subsequently, a post hoc analysis released at the 2021 European Hematology Association (EHA) Congress showed that even in patients who did not meet the primary endpoint at 24 weeks of rotercept, most patients could still benefit from continued treatment after 48 weeks of treatment.
The benefits include the reduction of blood transfusion volume and frequency, as well as the reduction of serum ferritin levels; and the follow-up data show that the blood transfusion burden of patients receiving long-term rotercept continued to decrease
.
Figure 3.
The reduction of blood transfusion burden between the rotercept treatment group and the placebo group In conclusion, the BELIEVE study confirmed that rotercept not only effectively reduces the blood transfusion burden in patients with transfusion-dependent β-thalassaemia, but also significantly reduces serum ferritin, which may It reduces the risk of organ damage and death caused by iron overload, and the benefits are long-term.
At the same time, the adverse events are within the acceptable range, and most of the adverse events are below grade 3 and are well tolerated
.
"Ze" Yao Xinsheng writes a new chapter The excellent curative effect of rotacept in international clinical research is obvious to all, and as an innovative drug just launched in China, the application value and clinical significance of rotacept in China are self-evident
.
As a subcutaneous injection drug, Libuloze only needs to be injected once every 3 weeks, which helps to reduce the frequency and time of medical treatment, blood transfusion and iron removal in patients with β-thalassemia, and effectively reduces the complications caused by blood transfusion and iron removal.
Physical, psychological and economic burden, greatly improve the quality of life of patients
.
In addition, for domestic clinical practice, the drug provides a brand-new TD β-thalassaemia treatment plan, filling the gap in the treatment field, and will also contribute to the implementation and practice of standardized diagnosis and treatment of thalassemia
.
In addition to TD β-thalassaemia, a breakthrough has been made in the application of red blood cell maturation agents in adult patients with non-transfusion-dependent (NTD) β-thalassemia
.
The results of the Phase II BEYOND study7 in patients with NTD β-thalassaemia were presented at the 2021 EHA Congress as LBA (Late-breaking Abstract), and 77.
1% of patients in the experimental group had higher hemoglobin levels during weeks 13-24 without blood transfusions.
Baseline increase of ≥1.
0 g/dL, while no patients in the placebo group met this primary endpoint (p<0.
0001); 89.
6% of patients in the trial group remained transfusion-free from 1 to 24 weeks versus 67.
3% in the placebo group (p=0.
0013)
.
Click to read: Professor Lai Yongrong's in-depth interpretation of the BEYOND study Based on this study, the marketing application of Librozer for the treatment of patients with NTD β-thalassaemia has been granted priority review status by the FDA
.
This also means that rottercept may soon benefit all beta-thalassemia patients
.
During the launch of Rottercept in China, it has witnessed the unremitting efforts of thousands of clinical, scientific research and related staff and the high hopes of many patients
.
The unique mechanism, excellent curative effect and huge potential constitute a solid "foundation" for rotacept in the field of β-thalassaemia treatment.
Build a building for innovative treatment of β-thalassemia in China to shield more β-thalassemia patients from wind and rain; at the same time, it helps to relieve the shortage of blood products in clinical practice and promote the balance of supply and demand of blood resources in the whole society
.
References: [1] Hematology Group of Pediatrics Branch of Chinese Medical Association, Editorial Board of "Chinese Journal of Pediatrics".
Guidelines for the diagnosis and treatment of β-thalassemia major (2017 edition) [J].
Chinese Journal of Pediatrics, 2018, 56(10 ): 6.
[2]https:// Xiangmin, Wang Lin, Yin Xiaolin, et al.
Operational Guidelines for Thalassaemia Prevention and Control[J ].
People’s Military Medical Press, 2011.
[5]Guerra A, Oikonomidou PR, Sinha S, et al.
Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemia.
Blood 2019 ;134:568-572.
[6]MD Cappellini, Viprakasit V, Taher AT, et al.
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia[J].
New England Journal of Medicine, 2020, 382 (13):1219-1231.
[7]Taher AT, Cappellini MD, Kattamis A et al.
The BEYOND study: results of a phase 2, double-blind, randomized, placebo-controlled multicenter study of luspatercept in adult patients with non -transfusion-dependent β-thalassemia.
2021 EHA, Abstract: S101.
Click "read the original text", we will make progress together
