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Bleeding and Clotting Disorder News & Archives

​Many Watching FDA for Ruling on Subcutaneous Formulation of Hemlibra

The FDA has set an October 4, 2018 deadline to potentially approve the use of Hemlibra for patients who do not have inhibitors. The medication could benefit hemophilia patients not only in a potentially longer period of time between treatment, but the medication can be administered subcutaneously. See full article at

​Liver Transplant Highly Benefits, but May Not Fully Cure, Hemophilia

A liver transplant may be life saving for hemophilia patients, and usually hemophilia symptoms disappear after surgery. However, a patient in Japan who had a liver transplant due to hepatitis C continued to have mild hemophilia. The report was published in the journal Haemophilia. See full article at

​Study Finds More Hemophilia Patients are Overweight & Obese

About a third of European and North American hemophilia patients are overweight or obese. This excess weight may be harmful to hemophilia patients by adding increased pressure on joints, making bleeds more likely. The review of published studies showed that over a ten year period overweight/obesity increased 20% in adults and 40% in children with hemophilia. See full article at

​Genentech Shares Positive Phase III Results for Hemlibra in NEJM

Genentech announced that data from the Phase III Haven 3 Study, which evaluated Hemlibra showed a significant reduction in bleeds in hemophilia A patients. The article was published in the New England​ Journal of Medicine and represents a major advancement in hemophilia research. See full article at

​FDA Approves Jivi, a New Hemophilia A Treatment

Bayer announced that the FDA has approved Jivi (BAY 94-9027) a new anti hemophilic factor treatment for prophylactic care of hemophilia A patients. Jivi has a twice weekly dosing and can be adjusted to less or more frequent dosing depending on bleeds. The medication is approved for adults and children over the age of 12. See full article at

​Catalyst Provides Update on Phase 2/3 Trial for Hemophilia with Inhibitors

The Catalyst Biosciences shared an update on their ongoing hemophilia phase 2/3 trial designed to treat patients with inhibitors. The update provides data on an additional two patients who completed the trial of the subcutaneous prophylactic factor treatment for both hemophilia​ A and B patients with inhibitors. See full article at

They Thought Hemophilia was a "Lifelong Thing." They May Be Wrong.

The New York Times takes a look at hemophilia patients who are living on the edge of the promise of gene therapy. The hope of a cure exists as trials exhibit positive numbers, yet those who are a part of the trials also have uncertainty and challenges learning to manage their care in the f​ace of success. See full article at​

Review Finds Multiple Factors Contribute to Inhibitors in Hemophilia

A review in "Thrombosis Research" found that there are no easy answers to the reason behind inhibitors that form in hemophilia patients. Several factors seem to contribute to the development of these difficult to manage complications to hemophilia patients. See full article at

Spark Updates Data for Hemophilia A Gene Therapy​ Trial

Spark Therapeutics provided preliminary phase 1/2 data for their SPK-8011, an investigational gene therapy to treat hemophilia A. The data showed that in the 12 participants there has been a 97% reduction in annualized bleeding rate and a 97% reduction in annualized infusion rate.  See full article at​​

Cost Analysis Compares Gene Therapy and Prophylaxis Care for Hemophilia A

A mathematical cost analysis of gene therapy was compared to prophylactic care and found that with current information and projected information, gene therapy is likely to be more cost effective in the treatment of severe hemophilia A. The analysis was done by the University of Pittsburgh and was published in Blood Advances.  See full article at​

Pfizer initiates Phase 3 Study for Hemophilia B Gene Therapy

Pfizer has announced that it has initiated a Phase 3 study to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy as a lead in study to evaluate a gene therapy treatment for hemophilia B. The study will evaluate fidanacogene elaparvovec, a potential one-time treatment option.  See full article at​

FDA Issues New Guidance for Advancing Gene Therapies, Including Hemophilia

The FDA issued a statement clarifying their efforts to advance the development of gene therapies. A policy of allowing gene therapies a faster track has allowed three gene therapy products to see approval in the past year. A draft for specific guidance for Human Gene Therapy for Hemophilia were made available and will be finalized after feedback. See full article at​

Study Finds Hemophilia Patients Still at Risk for Cardiovascular Disease

A recent study published in the journal Blood Advances showed that despite the general protection against the development of cardiovascular diseases that hemophilia patients usually enjoy, a risk remains to male patients over the age of 50. Findings show that hemophilia patients can still produce plaques in the arteries at similar rates as the general population.  See full article at​

NHF Collaborating with ASH, ISTH, and WFH to Create Guidelines on VWD Care

The National Hemophilia Foundation is collaborating with the American Society of Hematology (ASH), the International Society on Thrombosis and Haemostasis (ISTH) and the World Federation of Hemophilia (WFH) to create guidelines on the diagnosis and management of von Willebrand Disease (VWD). Persons with VWD or those caring for them are invited to take part in a survey to help development of questions and outcomes included in the guidelines. See full article and a link to the survey at

UniQure Enrolls First Patient in Phase 3 HOPE-B Study of AMT-061

UniQure announced that it has enrolled its first patient in their Phase 3 Hope-B pivotal study of AMT-061, an investigational AAV5-based gene therapy to treat severe and moderate hemophilia B. AMT-061 has been granted Breakthrough Therapy Designation by the FDA and this study is planning on enrolling approximately 50 adult patients to test the therapy.  See full article at​

Catalyst Biosciences Shares Update on their CB 2679 Program for Hemophilia B

Catalyst Biosciences provided an update on their ongoing Phase 1/2 trial investigating its next generation Factor IX candidate CB 2679d/ISU304 for the treatment of severe hemophilia B. The research is focusing on subcutaneous dosing of a potent treatment for hemophilia B.  See full article at​

Grifols Announces Recall of One Lot of Profilnine

Grifols Biologicals has announced a recall of one lot of Profilnine. The recall is being conducted as a precautionary measure. An incorrect volume of sterile water for injection was packaged with Profilnine lot A1PBB00072. Based on data, the manufacturer​ concludes no serious safety issues. See full article at ​

FDA Grants Priority Review to Emicizumab for Hemophilia A Without Inhibitors

The FDA has accepted a supplemental Biologics License Application (sBLA) and granted Priority Review to emicizumab-kxwh (Hemlibra) for adults and children with hemophilia A without factor VIII inhibitors. The decision is supported by data from Genentech's Haven 3 study showing a 96% reduction in treated bleeds.  See full article at​