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Long press the QR code to receive Pegcetacoplan (EMPAVELI®) developed by Apellis Pharmaceuticals, a C3 inhibitor, which was approved by the FDA in May 2021 for the treatment of paroxysmal nocturnal hemoglobinuria in adults [1].
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disease of hematopoietic stem cells caused by mutations in the PIG-A gene on somatic Xp22.
1.
The incidence of PNH is 1 to 1.
5 parts per million, the diagnosis age of patients is generally between 35 and 40 years, and the median survival time of patients diagnosed with PNH is 10 years [2,3].
The approval of Pegcetacoplan is based on the clinical results of a Phase III clinical trial (APL2-302; NCT03500549).
The study included 80 PNH patients who were treated with Pegcetacoplan and Eculizumab.
After 16 weeks, the severity of anemia was compared between the two groups.
The results showed that the change in hemoglobin level in the Pegcetacoplan group from baseline (+3.
84 g/dL) was superior to the Eculizumab active control group (-1.
47 g/dL) (p≤0.
0001); 35/41 (85%) of the patients in the Pegcetacoplan group There was no blood transfusion within 16 weeks, and 6/39 (15%) patients in the Eculizumab group did not have a blood transfusion within 16 weeks [4].For details of Pegcetacoplan and drugs targeting C3, see the picture below: (click to enlarge to view) You can also click the link below to get the corresponding PDF HD version: https://data.
pharmacodia.
com/v3/insight/#/ListPdf?tag=2 Reference materials 1.
Drug crossing data: https://data.
pharmacodia.
com 2.
ZCançadoRD, Araújo ADS, Sandes AF, et al.
Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria.
Hematology, Transfusion andCell Therapy.
2020 Jul.
DOI: 10.
1016/j.
htct.
2020.
06.
006.
3.
FDA official website https:// 4.
FDA Label Yaodu APP "points new gameplay" company enjoys the database overvalued permissions.
Talking about the top 10 global sales of drugs in the dry granulation technology in 2020.
Discussing the ups and downs of 20 years, where is the future of kinase inhibitors? Click "Read the original text" to keep abreast of industry trends
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disease of hematopoietic stem cells caused by mutations in the PIG-A gene on somatic Xp22.
1.
The incidence of PNH is 1 to 1.
5 parts per million, the diagnosis age of patients is generally between 35 and 40 years, and the median survival time of patients diagnosed with PNH is 10 years [2,3].
The approval of Pegcetacoplan is based on the clinical results of a Phase III clinical trial (APL2-302; NCT03500549).
The study included 80 PNH patients who were treated with Pegcetacoplan and Eculizumab.
After 16 weeks, the severity of anemia was compared between the two groups.
The results showed that the change in hemoglobin level in the Pegcetacoplan group from baseline (+3.
84 g/dL) was superior to the Eculizumab active control group (-1.
47 g/dL) (p≤0.
0001); 35/41 (85%) of the patients in the Pegcetacoplan group There was no blood transfusion within 16 weeks, and 6/39 (15%) patients in the Eculizumab group did not have a blood transfusion within 16 weeks [4].For details of Pegcetacoplan and drugs targeting C3, see the picture below: (click to enlarge to view) You can also click the link below to get the corresponding PDF HD version: https://data.
pharmacodia.
com/v3/insight/#/ListPdf?tag=2 Reference materials 1.
Drug crossing data: https://data.
pharmacodia.
com 2.
ZCançadoRD, Araújo ADS, Sandes AF, et al.
Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria.
Hematology, Transfusion andCell Therapy.
2020 Jul.
DOI: 10.
1016/j.
htct.
2020.
06.
006.
3.
FDA official website https:// 4.
FDA Label Yaodu APP "points new gameplay" company enjoys the database overvalued permissions.
Talking about the top 10 global sales of drugs in the dry granulation technology in 2020.
Discussing the ups and downs of 20 years, where is the future of kinase inhibitors? Click "Read the original text" to keep abreast of industry trends
