Nearly two-thirds of children with severe hemophilia A on preventive, or prophylactic, treatment with once-weekly Altuviiio (efanesoctocog alfa) in the yearlong XTEND-Kids study were free of bleeding episodes needing treatment. More than 80% in this Phase 3 clinical trial also were free of spontaneous bleedings and joint bleeds needing…
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AscellaHealth and Audaire Health together are offering on a hemophilia management program that aims to containing prescription costs and help patients better manage their treatment needs. The program leverages AscellaHealth’s network of specialty pharmacy partners and Audaire’s healthcare technology to offer pharmacy network design, therapy utilization management, and clinical…
Sangamo Therapeutics will regain full rights to develop and market giroctocogene fitelparvovec, a gene therapy candidate for adults with moderately severe to severe hemophilia A, after Pfizer decided to end its involvement in the program. Pfizer’s decision not to advance into regulatory submissions came despite positive…
The first patient in a Phase 1/2 clinical trial (“the Alta trial”) designed to assess safety and tolerability of SB-525 gene therapy has received treatment. Sangamo Therapeutics, in a collaboration with Pfizer, is developing  the SB-525 gene therapy to correct the Factor VIII (FVIII) defect in hemophilia A…
NovoSeven (eptacog alfa [activated]) is a bypassing agent that’s approved to treat bleeding episodes, including those occurring during surgery, in adults and children with hemophilia A or B who have developed inhibitors — neutralizing antibodies against certain clotting factors.
Dear pharmaceutical companies, I am speaking to every one of you who makes a factor replacement product for hemophilia. Thank you for developing more options and safer products for our community. Because of your research, we have products that greatly improve our lives. We…
Takeda has presented early data on the prevalence of — and a possible solution for — one of gene therapy’s main hurdles: the development of an immune reaction against the viral-based delivery vectors used in such therapies. The findings, presented at the 61st American Society of Hematology (ASH) Annual Meeting…
If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient compared with other preventive treatments, specifically Hemlibra. That’s according to a draft evidence report issued by pricing watchdog the Institute for Clinical and Economic Review (ICER). An independent non-profit…
Oxidation of clotting protein factor VIII (FVIII) increased the immune response to the replacement therapy in a mouse model of severe hemophilia A, a study found. Conducted by researchers in France, the study, “Oxidation of factor VIII increases its immunogenicity in mice with severe hemophilia…