April 7, 2021
A study, published in Haemophilia, found that continuous intravenous infusions of Advate immediately before and after major orthopedic surgery is as safe and effective as a standard, short, bolus infusions at preventing bleeds in men with moderate to severe hemophilia A.
March 31, 2021
It is unlikely that uniQure's AMT-061, an investigational gene therapy for hemophilia B, caused a case of hepatocellular carcinoma, a form of liver cancer. The tumor was found in one patient as a part of a five year long study and is likely a cause of genetic mutations and other risk factors.
March 12, 2021
Genentech shared two updates to their Hemlibra label that were approved by the FDA. The changes provide clarity for prescribers to consider the long half-life of the product when prescribing a different product after Hemlibra. The other update indicates that Hemlibra has not been studied in people with severe hepatic or renal impairment and congenital hemophilia A with and without Factor VIII inhibitors.
March 10, 2021
The US FDA has given regenerative medicine advanced therapy (RMAT) designation to Roctovian, a new gene therapy for hemophilia A in development by BioMarin. The designation allows treatments that fulfill an unmet need in serious, life-threatening conditions to move more quickly in approval processes.
February 24, 2021
Treatment with Octapharma's Nuwiq shows a low risk of developing inhibitors for those with hemophilia A who have previously been untreated. The clinical study was published in Thrombosis and Haemostasis.
February 19, 2021
The US FDA has given fast track designation to efanesoctocog alfa, an investigational Factor VIII replacement therapy to treat hemophilia A. Formerly known as BIVV001, the therapy is being developed by Bioverativ and Sobi.
February 17, 2021
Researchers in Vienna, Austria found that those with acquired hemophilia A that are treated with Hemlibra responded well in a small study published in Blood in January. Clinical trials on this group are needed, but the results showed the medication has potential to prevent bleeding, reduce side effects, and be cost effective.
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.
January 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.
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