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.

Data Finds Fitusiran Reduced Bleeds in Patients with Hemophilia A and B

November 15, 2021

Findings from a Phase 3 trial will be presented at the American Society of Hematology Annual meeting next month that show Fitusiran significantly reduced bleeds for patients with hemophilia A and B with inhibitors. 


FDA Puts Hold on Gene Therapy SB-525 Phase 3 Trial

November 10, 2021

The US FDA placed a clinical hold on the phase 3 trial evaluating SB-525, an investigational therapy for hemophilia A. The pause is intended to give the agency time to review changes to the protocols after unusually high factor levels were reported on some patients.


Small Study Finds Extended FIX Prophylactic Therapy May Not Ease Pain

November 5, 2021

A small real-world study out of Ireland showed that though extended half-life recombinant factor IX preventative treatment for severe hemophilia B provided less bleeding frequency, there was still a high reliance on pain medications. 


Long-Term Preventative Use of Esperoct Effective in Hemophilia A

October 18, 2021

Data from two Phase 3 clinical trials showed that long-term prophylactic treatment with a fixed dose of Esperoct (turoctocog alfa pegol) was effective in preventing and stopping bleeding in people with severe hemophilia A. The studies followed patients over a period of five years.


"Reverse Vaccination" May Help Prevent Immune Response

October 11, 2021

A newly designed platform based on the principle of "reverse vaccination" could be used to prevent the development of an immune response that blocks the effectiveness of treatments for hemophilia and other disorders. 


Study Finds Breakthrough Bleeds Likely for Hemophilia A Patients on Hemlibra

September 29, 2021

According to a new study using real-world data from Israel, most people with hemophilia A who are using Hemlibra will experience spontaneous or traumatic bleeds at some point. The study also indicated the risk of spontaneous bleeds were higher for older individuals. 


FDA Restores BeneFIX as Prophylactic for Children with Hemophilia B

September 15, 2021

The US FDA has restored its approval of BeneFIX as a routine prophylactic to treat children with hemophilia B. The medication was restricted to only adults while a different treatment retained market exclusivity until this month.


SerpinPC Prevents Bleeds in Hemophilia A and B Patients in Phase 2a Trial

September 13, 2021

Results from an early study of SerpinPC, a subcutaneously injected medication, showed reduced bleed rates and reduced spontaneous joint bleeds in both hemophilia A and B patients. The medication showed similar results for both hemophilia A and B patients and can be used with inhibitors. 


NHF and HFA Partner to Create "Together Project"

September 7, 2021

The National Hemophilia Foundation and the Hemophilia Federation of America released a statement announcing a new, joint initiative called the "Together Project". The partnership will unite resources and align their missions in an effort to support mental health for bleeding disorder patients.


Emicizumab (Hemlibra) May Improve Outcomes for Acquired Hemophilia A

August 6, 2021

A survey sent to hematologists suggest that emicizumab (Hemlibra) could improve outcomes for patients with acquired hemophilia A but it is underutilized in HTCs. The survey results were shared at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Annual Congress. 


Phase 2 Study Shows Monoclonal Antibody Marstacimab Appears Safe and Effective

August 4, 2021

According to a recent Phase 2 study, long-term treatment with the monoclonal antibody marstacimab was well tolerated and effective in patients with severe hemophilia. The study showed decreased annualized bleeding rates with no serious side effects.


Spark Therapeutics Shares Update for Gene Therapy SPK-8011 Trial

August 2, 2021

Spark Therapeutics recently updated data from an ongoing phase 1/2 clinical trial of their SPK-8011 gene therapy to treat hemophilia A. Results appear to show a 91.2% reduction in annualized bleed rate and a 97% reduction in annualized infusion rate after treatment. 

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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