January 29, 2021
An analysis published in the journal ClinicoEconomics and Outcomes Research evaluated the efficacy of Esperoct in preventing bleeds in previously treated patients with severe hemophilia A. Findings showed Esperoct's use as prophylaxis resulted in the fewest traumatic, spontaneous, and joint bleeds of four EHL treatments.
July 25, 2021
BioMarin announced positive updates from their ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for those with severe hemophilia A. The trial includes participants who have been observed for at least one year post treatment.
January 21, 2021
CSL Behring issued a voluntary recall of one batch of Mononine Coagulation Factor IX (Human), a plasma-derived product used to treat hemophilia B. Risk to patients is considered low. Contact your pharmacy if you have questions about this recall.
January 18, 2021
A study published in the journal Haemophilia shared that Eloctate can induce immune tolerance in people with Hemophilia A more quickly than other regimens. Immune tolerance induction (ITI) is the only strategy known to be effective for removing inhibitors.
January 13, 2021
Roctavian, an investigational gene therapy for hemophilia A patients, significantly reduced bleeding rates and the need for other treatments over a year according to data from a Phase 3 clinical trail.
December 28, 2020
The National Hemophilia Foundation shared a FAQ page with a wealth of information about the new vaccines available for COVID-19. The page addresses many questions people may have, especially focused on those with bleeding disorders, as well as resources for information about the vaccine.
December 20, 2020
The U.S. Food and Drug Administration announced a clinical hold on uniQure's Phase 3 Hope-B Trial of AMT-061 in severe or moderately severe hemophilia B patients. The hold follows a safety report relating to a possibly related serious adverse event associated with a preliminary diagnosis of HCC, a form of liver cancer in one patient.
December 8, 2020
Pfizer and Sangamo announced updated data for their phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with severe hemophilia A. The data was announced at the 62nd Annual American Society of Hematology meeting.
December 7, 2020
The US FDA granted fast track designation for marzeptacog alfa activated (MarzAA), a lab made version of clotting factor VIIa for the subcutaneous treatment of episodic bleeds in hemophilia A or B patients with inhibitors. MarzAA is moving into a Phase 3 trial.
November 20, 2020
A Phase 3 Trial for AMT-061, a gene therapy treatment for hemophilia B being developed by uniQure, showed increased factor IX activity and controlled bleeding while markedly reducing the need for other treatments.
November 11, 2020
A small study, published in Haemophilia recently, shows that patients who did passive stretching and also received manual therapy, reduced problems with joint bleeds.
November 3, 2020
A manuscript reviewing research from a survey of U.S. bleeding disorder patients who seek care from an HTC was published in Haemophilia outlining the positive reviews of their care.
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