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(Shanghai, China-May 7, 2021) Roche Pharmaceuticals China announced that its subsidiary Suyou Lile® (English trade name: HEMLIBRA®, chemical generic name: Eimexizumab) has been awarded by the National Medical Products Administration of China ( NMPA) is officially approved for the routine preventive treatment of adults and children with severe hemophilia A (congenital factor VIII deficiency, FVIII<1%) without coagulation factor VIII inhibitors to prevent or reduce bleeding Frequency of attacks.
This approval also means that Shuyou Lile® has become China's first and currently the only conventional preventive drug for hemophilia A with or without factor VIII inhibitors in the body at the same time.
As a bispecific monoclonal antibody, Suyou Lile® has the advantages of significantly reducing bleeding risk, long half-life, low injection frequency, and convenient administration.
It provides more flexible and effective treatment options for patients with hemophilia and reduces the disease of patients.
burden.
Frequent infusion is the biggest obstacle to prevention and treatment.
Hemophilia A urgently needs better treatment options.
Hemophilia is a hereditary coagulopathy.
Due to the lack of sufficient coagulation factor VIII in the blood, frequent and repeated bleeding occurs in the joints, muscles, and central nervous system of patients with hemophilia, which can lead to permanent joint damage and disability.
At present, there are about 136,000 patients in China, among which hemophilia A is the most common, accounting for about 80%-85% of all hemophilia [1].
In all patients with hemophilia A, there are no coagulation factor VIII inhibitors in about 70%-80% of severe and 90%-95% of moderate to mild patients.
[2] "Preventive treatment is one of the most important treatments for hemophilia A", Professor Yang Renchi, director of the Thrombosis Hemostasis Center of the Hospital of Hematology, Chinese Academy of Medical Sciences, said "By supplementing hemophilia patients with coagulation factors in advance, they can maintain At a certain level, it can reduce the risk of bleeding and maintain normal joint and muscle function.
Long-term standardized preventive treatment can even enable patients to achieve the goal of zero bleeding.
"In 2020, the World Federation of Hemophilia released the "Guidelines for the Management of Hemophilia"[3] clearly pointed out: preventive treatment is the standard treatment plan for patients with moderate to severe hemophilia, especially children, and preventive treatment should be carried out at all times.
In order to avoid bleeding.
However, at present, less than 10% of hemophilia patients in China have received preventive treatment.
The preventive treatment rate is much lower than the world average and the level of developed countries.
The disability rate of adult patients is caused by insufficient treatment.
As high as 70%[5].
"For a long time, the standard preventive treatment of hemophilia A requires intravenous infusion of FVIII 2-3 times a week, and the total number of injections per year is as high as 104-183 times.
The burden of treatment is self-evident.
Professor Zhao Yongqiang, director of the Department of Hematology, Peking Union Medical College Hospital, believes that “frequent intravenous injections not only aggravate the bleeding risk of hemophilia patients, but also become one of the important limiting factors for preventive treatment.
"Professor Wu Runhui, deputy director of the Hematology Oncology Center of Beijing Children's Hospital Affiliated to Capital Medical University, said, "The approval of the new indication for Shuyou Lile® is of far-reaching significance.
As an innovative drug for conventional preventive treatment that can be used for both adult and child patients, it greatly simplifies the treatment process while significantly reducing the risk of bleeding.
The administration method of subcutaneous injection once a week solves the problem of intravenous access and compliance especially for children, so that more Chinese children can return to school and grow up healthily.
"Effectiveness and safety are both important.
Suyoulile® preventive treatment makes "zero bleeding" possible.
The approval of the non-inhibitor indication for Suyoulile® is based on three key phase three clinical trials HAVEN 3, HAVEN 4 and HAVEN 5 The
results of the HAVEN 3[6] study showed that compared with patients who did not receive prophylactic treatment, adults aged 12 years and older who received emeicilizumab prophylaxis without factor VIII inhibitors and In adolescents, bleeding was reduced by 96% (p<0.
0001) and 97% (p<0.
0001), respectively. And compared with the no preventive treatment group (n=18), the bleeding-related endpoints showed a significant reduction in the results, and the safety was good.
The HAVEN 4 study showed that among adults and adolescents 12 years of age and older with or without FVIII inhibitors who were treated with Immexizumab prophylaxis, 56.
1% of patients experienced zero treatment bleeding, and 90.
2% of patients experienced 3 times or Treat less bleeding.
The updated data of the HAVEN 3 and HAVEN 4 studies prove that [7], for patients with hemophilia A with or without FVIII inhibitors, the prophylactic treatment of imicelizumab is compared with the previous on-demand or prophylactic coagulation factor VIII Compared with treatment, there is a clinically significant improvement in long-term health-related quality of life.
HAVEN 5 is the first phase III clinical trial of Iimexizumab focused on the Chinese population.
The results of the study showed that the bleeding risk of patients receiving immelizumab prophylaxis was significantly reduced by 96%.
The annual bleeding rate of patients with different dosing regimens was 1.
0, and the zero bleeding rate was more than 55%.
The overall well tolerated.
Serious adverse events occurred [8].
"Following the approval in 2018 in China for the routine preventive treatment of hemophilia A with coagulation factor VIII inhibitors, the addition of new indications for Shuyou Lile® in China will undoubtedly bring the majority of patients with type A The gospel also provides doctors with new treatment options.
" Professor Sun Jing, deputy director of the Department of Hematology, Nanfang Hospital, Southern Medical University, said, "Whether it is an inhibitor or a non-inhibitor patient, Immerizumab has shown excellent results.
Efficacy and safety results.
With its excellent clinical performance, Shuyou Lile® is expected to help more patients effectively prevent bleeding or reduce the frequency of bleeding, and eventually stay away from wheelchairs and even return to normal life, and have a free life with
" zero bleeding " .
" To meet the needs of patients, Roche joins hands with all walks of life to create a better future for "hemophilia".
As the first humanized bispecific monoclonal antibody in the field of hemophilia in the world in 20 years, it is currently the only hemophilia innovation that is subcutaneously injected once a week.
As a drug, Shuyou Lile® has been recommended by the "Guidelines for the Treatment of Hemophilia in China (2020 Edition)" as a non-factor product that can be used for preventive treatment, and has benefited more than 10,000 hemophilia patients worldwide. "In recent years, thanks to the continuous deepening of the reform of the drug review and approval system, more and more innovative drugs have been launched in China and abroad.
The " time difference" has been shortening.
This time, the new indication of Shuyou Lile® has passed the priority review and approval to speed up the market.
It is an excellent demonstration of the'China speed'.
" Zhou Hong, President of Roche Pharmaceuticals China, said, "Roche will always uphold the concept of'before the needs of patients', continue to develop innovative drugs in the field of hemophilia, and at the same time improve patients' awareness of these innovations.
The availability of medicines has benefited more Chinese patients.
"About Suyou Lile® (imicelizumab) is a humanized bispecific monoclonal antibody with dual specificity Sexual structure is like two arms.
It can bring together the proteins required to activate the natural coagulation cascade-activated coagulation factor IX and coagulation factor X, thereby restoring the coagulation process of patients with hemophilia A and allowing blood type A The goal of zero bleeding for friendly patients becomes possible.
Iimerizumab is China's first conventional preventive treatment drug approved for patients with hemophilia A and coagulation factor VIII inhibitors, and it is also the first preventive treatment drug that can be injected subcutaneously once a week.
Compared with the current alternative treatment methods, Immelizumab has the following advantages: the coagulation effect is not interfered by the eight-factor inhibitor, which can significantly reduce the annual bleeding rate and make zero bleeding possible; the half-life is as long as 30 days, and injection The frequency is reduced, and the effectiveness of preventive treatment is stable; subcutaneous injection is convenient to use, which can avoid the damage of long-term intravenous injection to blood vessels, making it possible for home treatment and self-injection.
About HAVEN 3 HAVEN 3 is a randomized, multi-center, open phase III clinical study to evaluate the efficacy, safety, and pharmacokinetics of Iimerizumab in non-inhibitory patients with hemophilia A over 12 years of age.
Kinetic characteristics.
The results of the HAVEN 3 study showed that compared with patients who did not receive preventive treatment, adults and adolescents aged 12 years and older who did not receive factor VIII inhibitors who received immexizumab preventive treatment each or every two weeks had bleeding, respectively.
Reduction of 96% (p<0.
0001) and 97% (p<0.
0001).
And compared with the no preventive treatment group (n=18), the bleeding-related endpoints showed a significant reduction in the results, and the safety was good.
About HAVEN 4 HAVEN 4 is a single-arm, multi-center, open-ended phase III clinical study to evaluate the efficacy of Iimexizumab in patients with hemophilia A who are 12 years of age and older with or without FVIII inhibitors.
Safety and pharmacokinetic characteristics.
The results of the HAVEN 4 study showed that among adults and adolescents 12 years of age and older with or without FVIII inhibitors who were treated with imitizumab every 4 weeks, 56.
1% of patients experienced zero treatment bleeding, and 90.
2% of patients Experience 3 or less treatment bleeding.
About HAVEN 5 HAVEN 5 is the first phase III clinical trial conducted in the Asia-Pacific region focusing on the efficacy and safety of Iimerizumab in the prevention and treatment of adults or adolescents in the Chinese population.
The results of the HAVEN 5 study showed that the bleeding risk of patients receiving imitizumab prophylaxis was significantly reduced by 96%.
The annual bleeding rate of patients with different dosage regimens was 1.
0, and the zero bleeding rate was more than 55%.
The overall well tolerated.
No serious adverse events occurred, demonstrating efficacy and safety results that are highly similar to other studies in the HAVEN series. References: [1] Thrombosis and Hemostasis Group of Hematology Branch of Chinese Medical Association, Chinese Hemophilia Collaborative Group.
Chinese Expert Consensus on Diagnosis and Treatment of Hemophilia (2017 Edition)[J].
Chinese Journal of Hematology, 2017, 38 (5 ): 364-370.
DOI: 10.
3760/cma.
j.
issn.
0253-2727.
2017.
05.
002.
[2] Thrombosis and Hemostasis Group, Hematology Branch of Chinese Medical Association, Chinese Hemophilia Collaborative Group, Hemophilia Treatment China Guidelines 2020 Edition, 2020, Chinese Journal of Hematology, Vol.
41 No.
4.
[3] WFH Guidelines for the Management of Hemophilia, 3rd Edition, available at https://news.
wfh.
org/sneak-peak-of -new-wfh-guidelines-for-the-management-of-hemophilia-3rd-edition-given-at-virtual-summit/[4] Qu Yanji, Yin Huan, Pang Yuanjie, etc.
Current status of treatment of hemophilia patients in Mainland China A systematic review of economic burden and economic burden.
Chinese Journal of Evidence-Based Medicine.
2013,13(02):182-189.
[5] Zhang L,Li H,Zhao H,et al.
Retrospective analysis of 1312 patients with haemophilia and related disorders in a single Chinese institute.
Haemophilia.
2003,9(6):696-702.
[6] FDA approves Roche's Hemlibra for haemophilia A without factor VIII inhibitors, available at -cor-2018-10-04c.
htm.
[7] ISTH Academy,2019,Emicizumab Treatment is Efficacious and Well Tolerated Long Term in Persons with Haemophilia A (PwHA) with or without FVIII Inhibitors: Pooled Data from Four HAVEN Studies, available at:https://academy.
isth.
org/isth/2019/melbourne/273812 /michael.
callaghan.
emicizumab.
treatment.
is.
efficacious.
and.
well.
tolerated.
long.
html%253Ff%253Dlisting%25253D4%25252Abrowseby%25253D8%25252Asortby%25253D2%25252Amedia%25253D3%25252Aspeaker%25253D675708.
[8] S Wang, et al.
ISTH 2020 [PB0957].
Stamp "read the original text" and we will make progress togetherhtml%253Ff%253Dlisting%25253D4%25252Abrowseby%25253D8%25252Asortby%25253D2%25252Amedia%25253D3%25252Aspeaker%25253D675708.
[8] S.
Wang, et al.
ISTH 2020 [PB0957].
Stamp "Read the original" and we will make progress togetherhtml%253Ff%253Dlisting%25253D4%25252Abrowseby%25253D8%25252Asortby%25253D2%25252Amedia%25253D3%25252Aspeaker%25253D675708.
[8] S.
Wang, et al.
ISTH 2020 [PB0957].
Stamp "Read the original" and we will make progress together
This approval also means that Shuyou Lile® has become China's first and currently the only conventional preventive drug for hemophilia A with or without factor VIII inhibitors in the body at the same time.
As a bispecific monoclonal antibody, Suyou Lile® has the advantages of significantly reducing bleeding risk, long half-life, low injection frequency, and convenient administration.
It provides more flexible and effective treatment options for patients with hemophilia and reduces the disease of patients.
burden.
Frequent infusion is the biggest obstacle to prevention and treatment.
Hemophilia A urgently needs better treatment options.
Hemophilia is a hereditary coagulopathy.
Due to the lack of sufficient coagulation factor VIII in the blood, frequent and repeated bleeding occurs in the joints, muscles, and central nervous system of patients with hemophilia, which can lead to permanent joint damage and disability.
At present, there are about 136,000 patients in China, among which hemophilia A is the most common, accounting for about 80%-85% of all hemophilia [1].
In all patients with hemophilia A, there are no coagulation factor VIII inhibitors in about 70%-80% of severe and 90%-95% of moderate to mild patients.
[2] "Preventive treatment is one of the most important treatments for hemophilia A", Professor Yang Renchi, director of the Thrombosis Hemostasis Center of the Hospital of Hematology, Chinese Academy of Medical Sciences, said "By supplementing hemophilia patients with coagulation factors in advance, they can maintain At a certain level, it can reduce the risk of bleeding and maintain normal joint and muscle function.
Long-term standardized preventive treatment can even enable patients to achieve the goal of zero bleeding.
"In 2020, the World Federation of Hemophilia released the "Guidelines for the Management of Hemophilia"[3] clearly pointed out: preventive treatment is the standard treatment plan for patients with moderate to severe hemophilia, especially children, and preventive treatment should be carried out at all times.
In order to avoid bleeding.
However, at present, less than 10% of hemophilia patients in China have received preventive treatment.
The preventive treatment rate is much lower than the world average and the level of developed countries.
The disability rate of adult patients is caused by insufficient treatment.
As high as 70%[5].
"For a long time, the standard preventive treatment of hemophilia A requires intravenous infusion of FVIII 2-3 times a week, and the total number of injections per year is as high as 104-183 times.
The burden of treatment is self-evident.
Professor Zhao Yongqiang, director of the Department of Hematology, Peking Union Medical College Hospital, believes that “frequent intravenous injections not only aggravate the bleeding risk of hemophilia patients, but also become one of the important limiting factors for preventive treatment.
"Professor Wu Runhui, deputy director of the Hematology Oncology Center of Beijing Children's Hospital Affiliated to Capital Medical University, said, "The approval of the new indication for Shuyou Lile® is of far-reaching significance.
As an innovative drug for conventional preventive treatment that can be used for both adult and child patients, it greatly simplifies the treatment process while significantly reducing the risk of bleeding.
The administration method of subcutaneous injection once a week solves the problem of intravenous access and compliance especially for children, so that more Chinese children can return to school and grow up healthily.
"Effectiveness and safety are both important.
Suyoulile® preventive treatment makes "zero bleeding" possible.
The approval of the non-inhibitor indication for Suyoulile® is based on three key phase three clinical trials HAVEN 3, HAVEN 4 and HAVEN 5 The
results of the HAVEN 3[6] study showed that compared with patients who did not receive prophylactic treatment, adults aged 12 years and older who received emeicilizumab prophylaxis without factor VIII inhibitors and In adolescents, bleeding was reduced by 96% (p<0.
0001) and 97% (p<0.
0001), respectively. And compared with the no preventive treatment group (n=18), the bleeding-related endpoints showed a significant reduction in the results, and the safety was good.
The HAVEN 4 study showed that among adults and adolescents 12 years of age and older with or without FVIII inhibitors who were treated with Immexizumab prophylaxis, 56.
1% of patients experienced zero treatment bleeding, and 90.
2% of patients experienced 3 times or Treat less bleeding.
The updated data of the HAVEN 3 and HAVEN 4 studies prove that [7], for patients with hemophilia A with or without FVIII inhibitors, the prophylactic treatment of imicelizumab is compared with the previous on-demand or prophylactic coagulation factor VIII Compared with treatment, there is a clinically significant improvement in long-term health-related quality of life.
HAVEN 5 is the first phase III clinical trial of Iimexizumab focused on the Chinese population.
The results of the study showed that the bleeding risk of patients receiving immelizumab prophylaxis was significantly reduced by 96%.
The annual bleeding rate of patients with different dosing regimens was 1.
0, and the zero bleeding rate was more than 55%.
The overall well tolerated.
Serious adverse events occurred [8].
"Following the approval in 2018 in China for the routine preventive treatment of hemophilia A with coagulation factor VIII inhibitors, the addition of new indications for Shuyou Lile® in China will undoubtedly bring the majority of patients with type A The gospel also provides doctors with new treatment options.
" Professor Sun Jing, deputy director of the Department of Hematology, Nanfang Hospital, Southern Medical University, said, "Whether it is an inhibitor or a non-inhibitor patient, Immerizumab has shown excellent results.
Efficacy and safety results.
With its excellent clinical performance, Shuyou Lile® is expected to help more patients effectively prevent bleeding or reduce the frequency of bleeding, and eventually stay away from wheelchairs and even return to normal life, and have a free life with
" zero bleeding " .
" To meet the needs of patients, Roche joins hands with all walks of life to create a better future for "hemophilia".
As the first humanized bispecific monoclonal antibody in the field of hemophilia in the world in 20 years, it is currently the only hemophilia innovation that is subcutaneously injected once a week.
As a drug, Shuyou Lile® has been recommended by the "Guidelines for the Treatment of Hemophilia in China (2020 Edition)" as a non-factor product that can be used for preventive treatment, and has benefited more than 10,000 hemophilia patients worldwide. "In recent years, thanks to the continuous deepening of the reform of the drug review and approval system, more and more innovative drugs have been launched in China and abroad.
The " time difference" has been shortening.
This time, the new indication of Shuyou Lile® has passed the priority review and approval to speed up the market.
It is an excellent demonstration of the'China speed'.
" Zhou Hong, President of Roche Pharmaceuticals China, said, "Roche will always uphold the concept of'before the needs of patients', continue to develop innovative drugs in the field of hemophilia, and at the same time improve patients' awareness of these innovations.
The availability of medicines has benefited more Chinese patients.
"About Suyou Lile® (imicelizumab) is a humanized bispecific monoclonal antibody with dual specificity Sexual structure is like two arms.
It can bring together the proteins required to activate the natural coagulation cascade-activated coagulation factor IX and coagulation factor X, thereby restoring the coagulation process of patients with hemophilia A and allowing blood type A The goal of zero bleeding for friendly patients becomes possible.
Iimerizumab is China's first conventional preventive treatment drug approved for patients with hemophilia A and coagulation factor VIII inhibitors, and it is also the first preventive treatment drug that can be injected subcutaneously once a week.
Compared with the current alternative treatment methods, Immelizumab has the following advantages: the coagulation effect is not interfered by the eight-factor inhibitor, which can significantly reduce the annual bleeding rate and make zero bleeding possible; the half-life is as long as 30 days, and injection The frequency is reduced, and the effectiveness of preventive treatment is stable; subcutaneous injection is convenient to use, which can avoid the damage of long-term intravenous injection to blood vessels, making it possible for home treatment and self-injection.
About HAVEN 3 HAVEN 3 is a randomized, multi-center, open phase III clinical study to evaluate the efficacy, safety, and pharmacokinetics of Iimerizumab in non-inhibitory patients with hemophilia A over 12 years of age.
Kinetic characteristics.
The results of the HAVEN 3 study showed that compared with patients who did not receive preventive treatment, adults and adolescents aged 12 years and older who did not receive factor VIII inhibitors who received immexizumab preventive treatment each or every two weeks had bleeding, respectively.
Reduction of 96% (p<0.
0001) and 97% (p<0.
0001).
And compared with the no preventive treatment group (n=18), the bleeding-related endpoints showed a significant reduction in the results, and the safety was good.
About HAVEN 4 HAVEN 4 is a single-arm, multi-center, open-ended phase III clinical study to evaluate the efficacy of Iimexizumab in patients with hemophilia A who are 12 years of age and older with or without FVIII inhibitors.
Safety and pharmacokinetic characteristics.
The results of the HAVEN 4 study showed that among adults and adolescents 12 years of age and older with or without FVIII inhibitors who were treated with imitizumab every 4 weeks, 56.
1% of patients experienced zero treatment bleeding, and 90.
2% of patients Experience 3 or less treatment bleeding.
About HAVEN 5 HAVEN 5 is the first phase III clinical trial conducted in the Asia-Pacific region focusing on the efficacy and safety of Iimerizumab in the prevention and treatment of adults or adolescents in the Chinese population.
The results of the HAVEN 5 study showed that the bleeding risk of patients receiving imitizumab prophylaxis was significantly reduced by 96%.
The annual bleeding rate of patients with different dosage regimens was 1.
0, and the zero bleeding rate was more than 55%.
The overall well tolerated.
No serious adverse events occurred, demonstrating efficacy and safety results that are highly similar to other studies in the HAVEN series. References: [1] Thrombosis and Hemostasis Group of Hematology Branch of Chinese Medical Association, Chinese Hemophilia Collaborative Group.
Chinese Expert Consensus on Diagnosis and Treatment of Hemophilia (2017 Edition)[J].
Chinese Journal of Hematology, 2017, 38 (5 ): 364-370.
DOI: 10.
3760/cma.
j.
issn.
0253-2727.
2017.
05.
002.
[2] Thrombosis and Hemostasis Group, Hematology Branch of Chinese Medical Association, Chinese Hemophilia Collaborative Group, Hemophilia Treatment China Guidelines 2020 Edition, 2020, Chinese Journal of Hematology, Vol.
41 No.
4.
[3] WFH Guidelines for the Management of Hemophilia, 3rd Edition, available at https://news.
wfh.
org/sneak-peak-of -new-wfh-guidelines-for-the-management-of-hemophilia-3rd-edition-given-at-virtual-summit/[4] Qu Yanji, Yin Huan, Pang Yuanjie, etc.
Current status of treatment of hemophilia patients in Mainland China A systematic review of economic burden and economic burden.
Chinese Journal of Evidence-Based Medicine.
2013,13(02):182-189.
[5] Zhang L,Li H,Zhao H,et al.
Retrospective analysis of 1312 patients with haemophilia and related disorders in a single Chinese institute.
Haemophilia.
2003,9(6):696-702.
[6] FDA approves Roche's Hemlibra for haemophilia A without factor VIII inhibitors, available at -cor-2018-10-04c.
htm.
[7] ISTH Academy,2019,Emicizumab Treatment is Efficacious and Well Tolerated Long Term in Persons with Haemophilia A (PwHA) with or without FVIII Inhibitors: Pooled Data from Four HAVEN Studies, available at:https://academy.
isth.
org/isth/2019/melbourne/273812 /michael.
callaghan.
emicizumab.
treatment.
is.
efficacious.
and.
well.
tolerated.
long.
html%253Ff%253Dlisting%25253D4%25252Abrowseby%25253D8%25252Asortby%25253D2%25252Amedia%25253D3%25252Aspeaker%25253D675708.
[8] S Wang, et al.
ISTH 2020 [PB0957].
Stamp "read the original text" and we will make progress togetherhtml%253Ff%253Dlisting%25253D4%25252Abrowseby%25253D8%25252Asortby%25253D2%25252Amedia%25253D3%25252Aspeaker%25253D675708.
[8] S.
Wang, et al.
ISTH 2020 [PB0957].
Stamp "Read the original" and we will make progress togetherhtml%253Ff%253Dlisting%25253D4%25252Abrowseby%25253D8%25252Asortby%25253D2%25252Amedia%25253D3%25252Aspeaker%25253D675708.
[8] S.
Wang, et al.
ISTH 2020 [PB0957].
Stamp "Read the original" and we will make progress together
