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Bleeding and Clotting Disorder News & Archives

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​

Octapharma Presents Data on Nuwiq for Hemophilia A Patients

Octapharma announced new data on the benefits of Nuwiq in patients with hemophilia A. The data was presented at a symposium at the WFH World Congress in Glasgow, Scotland. Their data focused on the approach of treatment, treating patients using the individual's PK profile to optimize their treatment plan. See full article at​

Spark and Pfizer Share Data from SPK-9001 Trial for Hemophilia B Patients

Spark Therapeutics and Pfizer announced data from 15 participants with hemophilia B showing persistent and sustained Factor IX levels with no serious side effects from their Phase 1/2 clinical trial of SPK-9001. The data showed dramatic reductions in bleeding and reduced annual bleeding rates by 98%. See full article at​

FDA to Speed Path to Approval for Some Gene Therapies-Starting w/Hemophilia

The FDA announced that it will alert companies that certain gene therapies in development can qualify for less arduous review. Specifically, gene therapies for hemophilia could be evaluated based on whether therapy increases proteins, regardless of whether it actually causes less bleeding. See full article at

Data from Hemlibra (emicizumab-kxwh) Trials Shared at WFH World Congress

Genentech announced full results from their Phase III Haven 3 study and their Phase III Haven 4 study. Both studies evaluated the emicizumab-kxwh prophylactic treatment. High numbers of those treated experienced zero bleeds. The positive results show overal efficacy and safety. See full article at​​

Bioverativ Shares Data on BIVV001 for Hemophilia A Patients

Bioverativ Presented Preliminary Phase 1/2a Data on BIVV001. The results showed that it demonstrated an unprecedented half-life of 37 hours with high factor VIII activity of 5.6% at seven days following a single low dose. The data was presented at the WFH 2018 World Congress held in Glasgow​, Scotland. See full ar​ticle at​

BioMarin Announces First Patient Dosed in Gene Therapy Study for Hemophilia A Patients with AAV5 Antibodies

BioMarin Pharmaceutical announced that it has dosed the first patient in a Phase 1/2 Study evaluating its investigational gene therapy valoctocogene roxaparvovec in severe hemophilia A patients with pre-existing AAV5 antibodies. The trial is the first that is testing to see how gene therapy can benefit those who have antibodies to the vector. See full article at​

Takeda Secures Takeover of Shire with $62 B Deal

Takada Pharmaceutical will pay $62 billion to acquire the rare disease biotech company Shire, in a deal that will create one of the largest drug makers​ by sales. The takeover was reached after Shire rejected four previous offers. A quarter of Shire's revenues in 2017 came from hemophilia medication. See full article at​

Study Suggests Prophylaxis Could Reduce Hospitalizations for VWD Patients

Researchers in Sweden and the United States published an article focused on potential correlations between patients with von Willebrand disease (VWD), hospitalizations and prophylaxis. The retrospective study investigated the frequency of hospital admittance to determine whether a prophylactic treatment regimen was associated with a reduction of hospitalizations. See full article

Sharon Funk Discusses PT and Bleeding Disorders in Ask the Expert Podcast

Our expert Physical Therapist, Sharon Funk, was interviewed in the Ask the Expert Podcast in April. She answered questions and shared her experience and insights on joint disease that she has gained throughout the years treating people with bleeding disorders. The 44 minute episode is available at​

FDA Grants Breakthrough Therapy Designation for Hemlibra

The FDA has granted Breakthrough Therapy Designation for Hemlibra (emicizumab-kxwh) for hemophilia A without factor VIII inhibitors. This designation was based on results from a Phase III trial. The treatment is being watched very carefully, both for its success and for safety issues. Five deaths were reported during the trials but were attributed to unrelated causes. See full article at​

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