In our Newsroom we share some recent headlines and links to articles that may be of interest to our bleeding and clotting disorder patients, families, and providers.

Study Shows Continuous Advate IV Effectively Prevented Bleeds in Surgery

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.


Investigation Shows That Gene Therapy AMT-061 Unlikely to Have Caused Liver Tumor

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.


Genentech Updates Hemlibra Labeling

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.


BioMarin's Roctavian, Gene Therapy for Hemophilia A, Gets FDA Regenerative Medicine Advanced Therapy Status

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.


Nuwiq Shows a Low Risk of Developing Inhibitors in Hemophilia A Trial

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


FDA puts Efanesoctocog Alfa on Fast Track Designation to treat Hemophilia A

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. 


Recent Study Shows Hemlibra Can Help Manage Acquired Hemophilia A

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. 


Study Shows Esperoct Highly Effective as Prophylaxis Treatment for Hemophilia A

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.


BioMarin Shares Positive Data for valactocogene roxaparvovec, Gene Therapy

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.


CSL Behring Issues Voluntary Recall of Mononine

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.


Study Suggests Eloctate Induces Faster Immune Tolerance than other Regimens

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.


Phase 3 Trial Shows Sustained Drop in Bleeding Rates for Roctavian, Gene Therapy for Hemophilia A

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. 

News Content Disclaimer:

Links to articles listed may take readers to websites separate and out of the control of the CU School of Medicine and the Hemophilia and Thrombosis Center. The CU School of Medicine and the Hemophilia and Thrombosis Center are not responsible for any errors, inaccuracies or opinions that may be expressed in any of the linked articles.

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