Iran announced in February that a pharmaceutical company based in the northeastern city of Mashhad had managed to produce Factor VIII
, becoming one of the few in the world to attain the knowledge needed for the production of the drug.
STASEY study is a phase IIIb clinical trial to evaluate the safety and tolerability of Hemlibra in people with hemophilia A with factor VIII
Updated data from the pooled HAVEN studies (HAVEN 1, HAVEN 2, HAVEN 3 and HAVEN 4; n=400), in people with hemophilia A of all ages with and without factor VIII
inhibitors, showed that a high proportion of patients experienced zero treated bleeds on Hemlibra, and that this was maintained over a median of 83 weeks.
"These data confirm that valoctocogene roxaparvovec has the potential to profoundly impact the lives of people with severe hemophilia A through a sustained reduction in bleeds and factor VIII
usage," said Hank Fuchs, M.D., President, Global Research and Development at BioMarin.
Conclusion: In conclusion, though development of factor VIII
inhibitors has not yet become major issue in our population and only 4.5% haemophiliaA patients developed inhibitors in present study.
In the intraindividual comparison involving 48 participants in group D, the annualized bleeding rate was 68 percent lower with emicizumab prophylaxis compared with the rate with previous factor VIII
The global hemophilia treatment market segmentation is based on drug type (antifibrinolytic agents, desmopressin, plasma derived coagulation factor concentrates - factor VIII
, factor IX, combination, recombinant coagulation factor concentrates - factor VIII
, factor IX, combination), indication type (acquired hemophilia, hemophilia a, hemophilia b, hemophilia c), and distribution channel (e-commerce, hospital pharmacies, retail pharmacies).
In one of our earlier studies we had reported 45 documented cases with 36 (81%) factor VIII
deficiency, 8 (17%) factor IX deficiency and one factor X deficiency case.
(2.) Mazurier C, Dieval J, JorieuxS, Delobel J, Goudemand M.A new von Willebrand factor (vWF) defect in a patient with factor VIII
(FVIII) deficiency but with normal levels and multimeric patterns of both plasma and platelet vWF.
The funds are earmarked to the HemAcure consortium with the goal to bring treatment of hemophilia A, also called factor VIII
deficiency--the most common form of hemophilia A--into the clinic for human trials.
The Bethesda test confirmed an elevated level of factor VIII
inhibitor at 6.16 IU/mL (normal 0.0 IU/mL) and a low level of factor VIII
at 0.02 IU/mL (normal 0.5-1.50 IU/mL), confirming a diagnosis of acquired hemophilia A.