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▎The content team editor of WuXi AppTec recently announced that Sanofi will announce the hemophilia RNAi therapy fitusiran jointly developed with Alnylam at this year’s American Society of Hematology (ASH) annual meeting to treat hemophilia A.
Results of two phase 3 clinical trials for hemophilia B and hemophilia
.
Hemophilia A and Hemophilia B are coagulation disorders caused by the patient's lack of coagulation factors VIII and IX, respectively
.
Fitusiran is an RNAi therapy that targets antithrombin (antithrombin).
It combines with RNA expressing antithrombin to reduce the production of antithrombin, thereby restoring the relationship between coagulation factors and anticoagulation factors.
Balance, achieve the effect of reducing bleeding events in patients with hemophilia
.
This innovative therapy only needs to be injected subcutaneously once a month.
Compared with conventional preventive coagulation factor injections, it provides a lot of convenience for patients to control the risk of bleeding
.
And it can treat patients with hemophilia A and B, regardless of whether they express inhibitors that inhibit exogenous coagulation factors in their bodies
.
▲The mechanism of action of Fitusiran (picture source: Reference [2]) The summary of the previous ASH annual meeting showed that fitusiran showed good activity in hemophilia patients with coagulation factor inhibitors, and 65% of the patients receiving treatment The annual bleeding rate is 0
.
The latest abstract of the ASH annual meeting showed that in patients who do not express inhibitors that inhibit exogenous coagulation factors in the body, a monthly dose of fitusiran significantly reduces the annual bleeding rate (ABR) of patients compared with receiving concentrated coagulation factors on demand.
.
The estimated ABR of any bleeding event that required treatment in the Fitusiran group was 3.
1, compared to 31.
0 in the control group, which reduced the ABR by 89.
9% compared to the control group
.
Among the patients in the Fitusiran group, 50.
6% of the patients did not have any bleeding that required treatment, compared to 5% in the control group
.
For detailed data, please see the following table: Picture source: Reference [1] In terms of safety, 78.
5% of fitusiran group patients and 12.
5% of control group patients had treatment-related adverse events
.
Two patients in the Fitusiran group terminated the trial due to serious adverse events
.
The researchers pointed out in the abstract that fitusiran met all the primary and secondary endpoints of this phase 3 clinical trial
.
It not only significantly reduces ABR, but also provides a meaningful improvement in the quality of life of patients
.
At present, researchers are improving the way of medication, by reducing the dosage and frequency of medication, increasing the benefit-risk ratio of fitusiran
.
Reference: [1] LBA-3 Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B ).
Retrieved November 23, 2021, from https://ash.
confex.
com/ash/2021/webprogram/Paper155018.
html[2] Development of a Pharmacokinetic-Pharmacodynamic (PK-PD) Model of Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia in Patients With and Without Inhibitors.
Retrieved June 19, 2020, from https:// : WuXi AppTec's content team focuses on introducing global biomedical health research progress
.
This article is for the purpose of information exchange only.
The opinions expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
.
This article is not a recommended treatment plan
.
If you need guidance on treatment plans, please go to a regular hospital for treatment
.
Results of two phase 3 clinical trials for hemophilia B and hemophilia
.
Hemophilia A and Hemophilia B are coagulation disorders caused by the patient's lack of coagulation factors VIII and IX, respectively
.
Fitusiran is an RNAi therapy that targets antithrombin (antithrombin).
It combines with RNA expressing antithrombin to reduce the production of antithrombin, thereby restoring the relationship between coagulation factors and anticoagulation factors.
Balance, achieve the effect of reducing bleeding events in patients with hemophilia
.
This innovative therapy only needs to be injected subcutaneously once a month.
Compared with conventional preventive coagulation factor injections, it provides a lot of convenience for patients to control the risk of bleeding
.
And it can treat patients with hemophilia A and B, regardless of whether they express inhibitors that inhibit exogenous coagulation factors in their bodies
.
▲The mechanism of action of Fitusiran (picture source: Reference [2]) The summary of the previous ASH annual meeting showed that fitusiran showed good activity in hemophilia patients with coagulation factor inhibitors, and 65% of the patients receiving treatment The annual bleeding rate is 0
.
The latest abstract of the ASH annual meeting showed that in patients who do not express inhibitors that inhibit exogenous coagulation factors in the body, a monthly dose of fitusiran significantly reduces the annual bleeding rate (ABR) of patients compared with receiving concentrated coagulation factors on demand.
.
The estimated ABR of any bleeding event that required treatment in the Fitusiran group was 3.
1, compared to 31.
0 in the control group, which reduced the ABR by 89.
9% compared to the control group
.
Among the patients in the Fitusiran group, 50.
6% of the patients did not have any bleeding that required treatment, compared to 5% in the control group
.
For detailed data, please see the following table: Picture source: Reference [1] In terms of safety, 78.
5% of fitusiran group patients and 12.
5% of control group patients had treatment-related adverse events
.
Two patients in the Fitusiran group terminated the trial due to serious adverse events
.
The researchers pointed out in the abstract that fitusiran met all the primary and secondary endpoints of this phase 3 clinical trial
.
It not only significantly reduces ABR, but also provides a meaningful improvement in the quality of life of patients
.
At present, researchers are improving the way of medication, by reducing the dosage and frequency of medication, increasing the benefit-risk ratio of fitusiran
.
Reference: [1] LBA-3 Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B ).
Retrieved November 23, 2021, from https://ash.
confex.
com/ash/2021/webprogram/Paper155018.
html[2] Development of a Pharmacokinetic-Pharmacodynamic (PK-PD) Model of Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia in Patients With and Without Inhibitors.
Retrieved June 19, 2020, from https:// : WuXi AppTec's content team focuses on introducing global biomedical health research progress
.
This article is for the purpose of information exchange only.
The opinions expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
.
This article is not a recommended treatment plan
.
If you need guidance on treatment plans, please go to a regular hospital for treatment
.
